{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T04:50:13Z","timestamp":1792644613968,"version":"4.5.0"},"reference-count":282,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2023,3,7]],"date-time":"2023-03-07T00:00:00Z","timestamp":1678147200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Viruses"],"abstract":"<jats:p>Viral vectors have been used for a broad spectrum of gene therapy for both acute and chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor, toxic, suicide and immunostimulatory genes, such as cytokines and chemokines, have been applied. Oncolytic viruses, which specifically replicate in and kill tumor cells, have provided tumor eradication, and even cure of cancers in animal models. In a broader meaning, vaccine development against infectious diseases and various cancers has been considered as a type of gene therapy. Especially in the case of COVID-19 vaccines, adenovirus-based vaccines such as ChAdOx1 nCoV-19 and Ad26.COV2.S have demonstrated excellent safety and vaccine efficacy in clinical trials, leading to Emergency Use Authorization in many countries. Viral vectors have shown great promise in the treatment of chronic diseases such as severe combined immunodeficiency (SCID), muscular dystrophy, hemophilia, \u03b2-thalassemia, and sickle cell disease (SCD). Proof-of-concept has been established in preclinical studies in various animal models. Clinical gene therapy trials have confirmed good safety, tolerability, and therapeutic efficacy. Viral-based drugs have been approved for cancer, hematological, metabolic, neurological, and ophthalmological diseases as well as for vaccines. For example, the adenovirus-based drug Gendicine\u00ae for non-small-cell lung cancer, the reovirus-based drug Reolysin\u00ae for ovarian cancer, the oncolytic HSV T-VEC for melanoma, lentivirus-based treatment of ADA-SCID disease, and the rhabdovirus-based vaccine Ervebo against Ebola virus disease have been approved for human use.<\/jats:p>","DOI":"10.3390\/v15030698","type":"journal-article","created":{"date-parts":[[2023,3,8]],"date-time":"2023-03-08T03:29:05Z","timestamp":1678246145000},"page":"698","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":189,"title":["Viral Vectors in Gene Therapy: Where Do We Stand in 2023?"],"prefix":"10.3390","volume":"15","author":[{"given":"Kenneth","family":"Lundstrom","sequence":"first","affiliation":[{"name":"PanTherapeutics, CH1095 Lutry, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,3,7]]},"reference":[{"key":"ref_1","unstructured":"Lundstrom, K. (2015). Novel Approaches and Strategies for Biologics, Vaccines and CancerTherapies, Elsevier."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1007\/s00439-013-1288-1","article-title":"Silencing human genetic diseases with oligonucleotide-based therapies","volume":"132","author":"Wright","year":"2013","journal-title":"Hum. Genet."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1146\/annurev-pharmtox-010715-103633","article-title":"RNA interference (RNAi)-based therapeutics: Delivering on the promise?","volume":"56","author":"Bobbin","year":"2016","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"ref_4","unstructured":"Ramirez-Montagut, T. (2015). Novel Approaches and Strategies for Biologics, Vaccines and Cancer Therapies, Elsevier."},{"key":"ref_5","doi-asserted-by":"crossref","unstructured":"Anurogo, D., Yuli Prasetyo Budi, N., Thi Ngo, M.H., Huang, Y.-H., and Pawitanm, J.A. (2021). Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing. Int. J. Mol. Sci., 22.","DOI":"10.3390\/ijms22126275"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1002\/hon.2591","article-title":"CAR-T cell therapy: Full speed ahead","volume":"37","author":"Sermer","year":"2019","journal-title":"Hematol. Oncol."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: A review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Delivery"},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1172\/JCI35700","article-title":"Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1","volume":"118","author":"Garrigue","year":"2008","journal-title":"J. Clin. Investig."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1016\/j.ymgme.2003.08.016","article-title":"Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer","volume":"80","author":"Raper","year":"2003","journal-title":"Mol. Genet. Metab."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1038\/s41392-021-00487-6","article-title":"Viral vector platforms within the gene therapy landscape","volume":"6","author":"Bulcha","year":"2021","journal-title":"Signal Transd. Targeted Ther."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"453","DOI":"10.2174\/15665232113136660006","article-title":"Biosafety challenges for use of lentiviral vectors in gene therapy","volume":"13","author":"Rothe","year":"2013","journal-title":"Curr. Gene Ther."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"1373","DOI":"10.1111\/cas.13027","article-title":"Oncolytic virus therapy: A new era of cancer treatment at dawn","volume":"107","author":"Fukuhara","year":"2016","journal-title":"Cancer Sci."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1016\/j.ymthe.2004.07.013","article-title":"Adenoviruses as vaccine vectors","volume":"10","author":"Tatsis","year":"2004","journal-title":"Mol. Ther."},{"key":"ref_14","first-page":"e52894","article-title":"Cloning and Large-Scale Production of High-Capacity Adenoviral Vectors Based on the Human Adenovirus Type 5","volume":"107","author":"Zhang","year":"2016","journal-title":"J. Vis. Exp."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1089\/hum.2013.2527","article-title":"Adenovirus: The First Effective in Vivo Gene Delivery Vector","volume":"25","author":"Crystal","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1452","DOI":"10.1161\/01.ATV.20.6.1452","article-title":"Second-generation adenovirus vectors do not prevent rapid loss of transgene expression and vector DNA from the arterial wall","volume":"20","author":"Wen","year":"2000","journal-title":"Arterioscler. Thromb. Vasc. Biol."},{"key":"ref_17","doi-asserted-by":"crossref","unstructured":"Ricobaraza, A., Gonzalez-Aparicio, M., Mora-Jimenez, L., Lumbreras, S., and Hernadez-Alcoceba, R. (2020). High-Capacity Adenovirus Vectors: Expanding the Scope of Gene Therapy. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21103643"},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"67","DOI":"10.2174\/156652312800099599","article-title":"Biodistribution and safety assessment of bladder cancer specific oncolytic adenovirus in subcutaneous xenografts tumor model in nude mice","volume":"12","author":"Wang","year":"2012","journal-title":"Curr. Gene Ther."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"2383","DOI":"10.1159\/000475909","article-title":"Engineering the rapid adenovirus production and amplification (RAPA) cell line to expedite the generation of recombinant adenoviruses","volume":"41","author":"Wei","year":"2017","journal-title":"Cell Physiol. Biochem."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"7689","DOI":"10.1128\/JVI.77.13.7689-7695.2003","article-title":"Episomal Persitence of Recombinant Adenoviral Vector Genomes during the Cell Cycle In Vivo","volume":"77","author":"Ehrhardt","year":"2003","journal-title":"J. Virol."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1089\/hum.2013.071","article-title":"Transgene expression up to 7 years in nonhuman primates following hepatic transduction with helper-dependent adenoviral vectors","volume":"24","author":"Ng","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1146\/annurev-virology-031413-085355","article-title":"AAV-mediated gene therapy for research and therapeutic purposes","volume":"1","author":"Samulski","year":"2014","journal-title":"Annu. Rev. Virol."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"9933","DOI":"10.1128\/JVI.79.15.9933-9944.2005","article-title":"Packaging capacity of adeno-associated virus serotypes: Impact of larger genomes on infectivity and postentry steps","volume":"79","author":"Grieger","year":"2005","journal-title":"J. Virol."},{"key":"ref_24","first-page":"611","article-title":"Adeno-associated virus (AAV) dual vector strategies for gene therapy encoding large transgenes","volume":"90","author":"McClements","year":"2017","journal-title":"Yale J. Biol. Med."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"2653","DOI":"10.2741\/2872","article-title":"Cancer gene therapy using adeno-associated virus vectors","volume":"13","author":"Park","year":"2008","journal-title":"Front. Biosci."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: Overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"2019","DOI":"10.1182\/bloodadvances.2017010181","article-title":"Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors","volume":"1","author":"Meliani","year":"2017","journal-title":"Blood Adv."},{"key":"ref_28","first-page":"442","article-title":"Adeno-associated virus integration","volume":"11","author":"Deyle","year":"2009","journal-title":"Curr. Opin. Mol. Ther."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"1902","DOI":"10.1038\/mt.2012.157","article-title":"AAV Vectors Containing rDNA Homology Increased Chromosomal Integration and Transgene Persistence","volume":"20","author":"Wang","year":"2012","journal-title":"Mol. Ther."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"e01701-19","DOI":"10.1128\/JVI.01701-19","article-title":"Herpes Simplex Virus Latency Is Noisier The Closer We Look","volume":"94","author":"Singh","year":"2020","journal-title":"J. Virol."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"445","DOI":"10.2174\/156652305774329285","article-title":"HSV-1 derived recombinant and amplicon vectors for gene transfer and gene therapy","volume":"5","author":"Epstein","year":"2005","journal-title":"Curr. Gene Ther."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"12100","DOI":"10.1128\/JVI.01169-10","article-title":"Herpesviruses and chromosomal integration","volume":"84","author":"Morisette","year":"2010","journal-title":"J. Virol."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"33","DOI":"10.3109\/13550280009006380","article-title":"A multi-mutant herpes simplex virus vector has minimal cytotoxic effects on the distribution of filamentous actin, alpha-actinin and a glutamate receptor in differentiated PC-12 cells","volume":"6","author":"Holmes","year":"2000","journal-title":"J. Neurovirol."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"303","DOI":"10.2174\/156652306777592081","article-title":"DNA-based methods to prepare helper viurs-free herpes amplicon vectors and versatile design of amplicon vector plasmids","volume":"6","author":"Kasai","year":"2006","journal-title":"Curr. Gene Ther."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"6358","DOI":"10.1128\/jvi.70.9.6358-6369.1996","article-title":"Prolonged gene expression and cell surviavl after unfection by a herpes simplex virus mutant defective in the immediate-early early genes encoding ICP4, ICP27, and ICP22","volume":"70","author":"Wu","year":"1996","journal-title":"J. Virol."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1186\/1743-422X-10-241","article-title":"Micro-RNA-145 regulates oncolytic herpes simplex virus-1 for selective killing of human non-small lung cancer cells","volume":"10","author":"Li","year":"2013","journal-title":"Virol. J."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1006\/mthe.2001.0294","article-title":"Improved helper virus-free packaging system for HSV amplicon vectors using an ICP27-deleted, oversized HSV-1 DNA in a bacterial artificial chromosome","volume":"3","author":"Saeki","year":"2001","journal-title":"Mol. Ther."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1007\/978-3-319-42934-2_2","article-title":"Retroviral vectors for cancer gene therapy","volume":"209","author":"Schambach","year":"2016","journal-title":"Curr. Strat. Cancer Gene Ther."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"12730","DOI":"10.1021\/acs.chemrev.6b00125","article-title":"Retroviral DNA Integration","volume":"116","author":"Lesbats","year":"2016","journal-title":"Chem. Rev."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"1407","DOI":"10.1056\/NEJMoa1404588","article-title":"A modified \u03b3-retrovirus vector for X-linked severe combined immunodeficiency","volume":"371","author":"Pai","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1182\/blood.2021012300","article-title":"Built to last: Gene therapy for ADA SCID","volume":"138","author":"Pai","year":"2021","journal-title":"Blood"},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"1304","DOI":"10.1182\/blood.2020010260","article-title":"Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency","volume":"138","author":"Reinhardt","year":"2021","journal-title":"Blood"},{"key":"ref_43","first-page":"3635","article-title":"Gene therapies for primary immune deficiencies","volume":"120","author":"Kohn","year":"2021","journal-title":"Front. Immunol."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1016\/j.virol.2019.02.014","article-title":"Rapid establishment of stable retroviral packaging cells and recombinant susceptible target cell lines employing novel transposon vectors derived from Sleeping Beauty","volume":"531","author":"Berg","year":"2019","journal-title":"Virology"},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1038\/83324","article-title":"Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics","volume":"7","author":"Kay","year":"2001","journal-title":"Nat. Med."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"419","DOI":"10.2174\/156652308786848021","article-title":"Mechanisms governing lentivirus integration site selection","volume":"8","author":"Ciuffi","year":"2008","journal-title":"Curr. Gene Ther."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1089\/hum.2009.134","article-title":"Modification of integration site preferences of an HIV-1-based vector by expression of a novel synthetic protein","volume":"2","author":"Silvers","year":"2010","journal-title":"Hum. Gene Ther."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"1863","DOI":"10.1089\/10430340050129486","article-title":"Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system","volume":"11","author":"Nakajima","year":"2000","journal-title":"Hum. Gene Ther."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"2684","DOI":"10.3390\/v4112684","article-title":"Clinical aspects of feline retroviruses: A review","volume":"4","author":"Hartmann","year":"2012","journal-title":"Viruses"},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1038\/sj.gt.3300768","article-title":"Gene transfer vectors derived from equine infectious anemia virus","volume":"5","author":"Olsen","year":"1998","journal-title":"Gene Ther."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"e2000017","DOI":"10.1002\/biot.202000017","article-title":"Progress and Perspectives in the Development of Lentiviral Vector Producer Cells","volume":"16","author":"Ferreira","year":"2021","journal-title":"Biotechnol. J."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"491","DOI":"10.1128\/mr.58.3.491-562.1994","article-title":"The alphaviruses; gene expression, replication and evolution","volume":"58","author":"Strauss","year":"1994","journal-title":"Microbiol. Rev."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1002\/0471142301.ns0422s57","article-title":"Alphaviruses: Semliki Forest Virus and Sindbis Virus Vectors for Gene Transfer into Neurons","volume":"57","author":"Ehrengruber","year":"2011","journal-title":"Curr. Protocols Neurosci."},{"key":"ref_54","doi-asserted-by":"crossref","unstructured":"Lundstrom, K. (2020). Self-amplifying RNA viruses as RNA vaccines. Int. J. Mol. Sci., 2020.","DOI":"10.3390\/ijms21145130"},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"1356","DOI":"10.1038\/nbt1291-1356","article-title":"A new generation of animal cell expression vectors based on the Semliki Forest virus replicon","volume":"9","author":"Garoff","year":"1991","journal-title":"Biotechnology"},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"1188","DOI":"10.1126\/science.2922607","article-title":"Sindbis virus: An efficient, broad host range vector for gene expression in animal cells","volume":"243","author":"Xiong","year":"1989","journal-title":"Science"},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1016\/0042-6822(89)90526-6","article-title":"In vitro synthesis of infectious Venezuelan equine encephalitis virus RNA from a cDNA clone: Analysis of a viable deletion mutant","volume":"171","author":"Davis","year":"1989","journal-title":"Virology"},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"4783","DOI":"10.1038\/s41388-021-01869-4","article-title":"Necroptotic virotherapy of oncolytic alphavirus M1 cooperated with Doxorubicin displays promising therapeutic efficacy in TNBC","volume":"40","author":"Zhang","year":"2021","journal-title":"Oncogene"},{"key":"ref_59","doi-asserted-by":"crossref","unstructured":"Heikkil\u00e4, J.E., V\u00e4h\u00e4-Koskela, M.J., Ruotsalainen, J.J., Martikainen, M.W., Stanford, M.M., McCart, J.A., Bell, J.C., and Hinkkanen, A.E. (2010). Intravenously administered alphavirus vector VA7 eradicates orthotopic human glioma xenografts in nude mice. PLoS ONE, 5.","DOI":"10.1371\/journal.pone.0008603"},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1517\/14712598.6.2.135","article-title":"Kunjin virus replicons: An RNA-based, non-cytopathic viral vector system for protein production, vaccine and gene therapy applications","volume":"6","author":"Pijlman","year":"2006","journal-title":"Exp. Opin. Biol. Ther."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"11605","DOI":"10.1128\/JVI.78.21.11605-11614.2004","article-title":"Trans-packaged West Nile virus-like particles: Infectious properties in vitro and in infected mosquito vectors","volume":"78","author":"Scholle","year":"2004","journal-title":"J. Virol."},{"key":"ref_62","doi-asserted-by":"crossref","unstructured":"Pang, X., Zhang, M., and Dayton, A.I. (2001). Development of dengue virus type 2 replicons capable of prolonged expression in host cells. BMC Microbiol., 1.","DOI":"10.1186\/1471-2180-1-28"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"8924","DOI":"10.1128\/JVI.77.16.8924-8933.2003","article-title":"Incorporation of tick-borne encephalitis virus replicons into virus-like particles by a packaging cell line","volume":"77","author":"Gherke","year":"2003","journal-title":"J. Virol."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1016\/j.virol.2004.10.034","article-title":"Construction and applications of yellow fever virus replicons","volume":"331","author":"Jones","year":"2005","journal-title":"Virology"},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"2843","DOI":"10.1084\/jem.20171093","article-title":"Zika virus has oncolytic activity against glioblastoma stem cells","volume":"214","author":"Zhu","year":"2017","journal-title":"J. Exp. Med."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"5967","DOI":"10.1128\/JVI.72.7.5967-5977.1998","article-title":"Encapsidation of the flavivirus Kunjin replicon RNA by using a complementation system providing Kunjin virus structural proteins in trans","volume":"72","author":"Khromykh","year":"1998","journal-title":"J. Virol."},{"key":"ref_67","doi-asserted-by":"crossref","unstructured":"Apostolopoulos, V. (2016). Vaccine delivery methods into the future. Vaccine, 4.","DOI":"10.3390\/vaccines4020009"},{"key":"ref_68","first-page":"1","article-title":"Self-replicating vehicles based on negative strand RNA viruses","volume":"15","author":"Lundstrom","year":"2022","journal-title":"Cancer Gene Ther."},{"key":"ref_69","first-page":"129","article-title":"Reverse genetics of measles virus and resulting multivalent recombinant vaccines: Applications of recombinant measles virus","volume":"329","author":"Billeter","year":"2009","journal-title":"Curr. Top. Microbiol. Immunol."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1016\/j.ijid.2016.02.722","article-title":"Engineering of measles virus to target cancer cells, an attempt","volume":"45","author":"Lal","year":"2016","journal-title":"Intl. J. Infect. Dis."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"199","DOI":"10.3892\/or.2012.2109","article-title":"Live attenuated measles virus vaccine induces apoptosis and promotes tumor regression in lung cancer","volume":"29","author":"Zhao","year":"2013","journal-title":"Oncol. Rep."},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"387362","DOI":"10.1155\/2013\/387362","article-title":"Natural oncolytic activity of live-attenuated measles virus against human lung and colorectal adenocarcinomas","volume":"2013","author":"Boisgerault","year":"2013","journal-title":"Biomed. Res. Int."},{"key":"ref_73","first-page":"165","article-title":"Recombinant rhabdoviruses: Vectors for vaccine development and gene therapy","volume":"292","author":"Finke","year":"2005","journal-title":"Curr. Top. Microbiol. Immunol."},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1007\/978-1-4939-6964-7_12","article-title":"Rapid Reverse Genetics Systems for Rhabdovirus From Forward to Reverse and Back Again","volume":"1602","author":"Nolden","year":"2017","journal-title":"Methods Mol. Biol."},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"1786","DOI":"10.1002\/ijc.31556","article-title":"Oncolytic activity of the rhabdovirus VSV-GP against prostate cancer","volume":"143","author":"Urbiola","year":"2018","journal-title":"Int. J. Cancer"},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"1257","DOI":"10.1002\/ijc.30813","article-title":"Oncolytic Maraba Virus MG1 as a Treatment for Sarcoma","volume":"141","author":"Selman","year":"2017","journal-title":"Int. J. Cancer"},{"key":"ref_77","doi-asserted-by":"crossref","first-page":"613","DOI":"10.1111\/j.1348-0421.2003.tb03424.x","article-title":"Improved recovery of rabies virus from cloned cDNA using a vaccinia virus-free reverse genetics system","volume":"47","author":"Ito","year":"2003","journal-title":"Microbiol. Immunol."},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1016\/j.virusres.2014.02.016","article-title":"Newcastle disease virus: Current status and our understanding","volume":"184","author":"Ganar","year":"2014","journal-title":"Virus Res."},{"key":"ref_79","first-page":"945","article-title":"Antitumor effects of Newcastle disease virus in vivo: Local versus systemic effects","volume":"18","author":"Schirrmacher","year":"2001","journal-title":"Int. J. Oncol."},{"key":"ref_80","first-page":"161","article-title":"Poxviruses as vectors for cancer immunotherapy","volume":"6","author":"Kwak","year":"2003","journal-title":"Curr. Opin. Drug Discov. Devel."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"1001","DOI":"10.1038\/sj.cgt.7700549","article-title":"Development of a replication-selective oncolytic proxvirus for the treatment of human cancers","volume":"9","author":"Zeh","year":"2002","journal-title":"Cancer Gene Ther."},{"key":"ref_82","doi-asserted-by":"crossref","first-page":"47","DOI":"10.2147\/OV.S56322","article-title":"Applications of coxsackievirus A21 in oncology","volume":"3","author":"Bradley","year":"2014","journal-title":"Oncol. Virother."},{"key":"ref_83","doi-asserted-by":"crossref","first-page":"410","DOI":"10.4161\/hv.7.4.14422","article-title":"Application of attenuated coxsackievirus B3 as viral vector system for vaccines and gene therapy","volume":"7","author":"Kim","year":"2011","journal-title":"Hum. Vaccin."},{"key":"ref_84","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1080\/135502802317247776","article-title":"Expression of brain-derived neurotrophic factor in the central nervous system of mice using a poliovirus-based vector","volume":"8","author":"Jia","year":"2002","journal-title":"J. Neurovirol."},{"key":"ref_85","first-page":"69","article-title":"Reovirus in cancer therapy: An evidence-based review","volume":"3","author":"Clements","year":"2014","journal-title":"Oncol. Virother."},{"key":"ref_86","first-page":"457","article-title":"Reovirus\u2014Possible therapy of cancer","volume":"53","author":"Figova","year":"2006","journal-title":"Neoplasma"},{"key":"ref_87","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1158\/1535-7163.MCT-15-0695","article-title":"Strategic Combinations: The Future of Oncolytic Virotherapy with Reovirus","volume":"15","author":"Zhao","year":"2016","journal-title":"Mol. Cancer Ther."},{"key":"ref_88","doi-asserted-by":"crossref","first-page":"e728","DOI":"10.1038\/cddis.2013.259","article-title":"Reolysin is a novel reovirus-based agent that induces endoplasmic reticular stress-mediated apoptosis in pancreatic cancer","volume":"4","author":"Carew","year":"2013","journal-title":"Cell Death Dis."},{"key":"ref_89","doi-asserted-by":"crossref","first-page":"451","DOI":"10.2174\/1389201043376670","article-title":"SV40 pseudovirions as highly efficient vectors for gene transfer and their potential application in cancer therapy","volume":"5","author":"Gottesman","year":"2004","journal-title":"Curr. Pharm. Biotechnol."},{"key":"ref_90","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1016\/j.omtm.2017.06.007","article-title":"Generation of a Vero-based packaging cell line to produce SV40 gene delivery vectors for use in clinical gene therapy studies","volume":"6","author":"Toscano","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_91","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1038\/sj.cgt.7700987","article-title":"Replication-deficient rSV40 mediate pancreatic gene transfer and long-term inhibition of tumor growth","volume":"14","author":"Cordelier","year":"2007","journal-title":"Cancer Gene Ther."},{"key":"ref_92","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1007\/s11262-016-1313-2","article-title":"A recombinant rabies virus carrying GFP between N and P affects viral transcription in vitro","volume":"52","author":"Luo","year":"2016","journal-title":"Virus Genes"},{"key":"ref_93","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1016\/j.virusres.2012.11.007","article-title":"Genetically modified VSV(NJ) vector is capable of accommodating a large foreign gene insert and allows high level gene expression","volume":"171","author":"An","year":"2013","journal-title":"Virus Res."},{"key":"ref_94","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/mt.2013.249","article-title":"Maraba virus as a potent oncolytic vaccine vector","volume":"22","author":"Pol","year":"2014","journal-title":"Mol. Ther."},{"key":"ref_95","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1016\/0022-4804(92)90310-V","article-title":"Newcastle disease virus selectively kills human tumor cells","volume":"52","author":"Reichard","year":"1992","journal-title":"J. Surg. Res."},{"key":"ref_96","doi-asserted-by":"crossref","first-page":"5343","DOI":"10.1128\/JVI.00136-16","article-title":"Genetic modification of oncolytic Newcastle disease virus for cancer therapy","volume":"90","author":"Cheng","year":"2016","journal-title":"J. Virol."},{"key":"ref_97","unstructured":"Lundstrom, K. (2022). The Biological Role of a Virus. Advances in Environmental Microbiology, Springer Nature."},{"key":"ref_98","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1016\/S0147-9571(03)00019-5","article-title":"Poxviruses as vaccine vectors","volume":"26","author":"Pastoret","year":"2003","journal-title":"Comp. Immunol. Microbiol. Infect. Dis."},{"key":"ref_99","doi-asserted-by":"crossref","first-page":"2924","DOI":"10.1158\/1535-7163.MCT-10-0590","article-title":"Reovirus virotherapy overrides tumor antigen presentation evasion and promotes protective antitumor immunity","volume":"9","author":"Gujar","year":"2010","journal-title":"Mol. Cancer. Ther."},{"key":"ref_100","doi-asserted-by":"crossref","first-page":"3027","DOI":"10.1182\/blood-2005-10-4114","article-title":"Tumor vascular targeting therapy with viral vectors","volume":"107","author":"Liu","year":"2006","journal-title":"Blood"},{"key":"ref_101","doi-asserted-by":"crossref","first-page":"605380","DOI":"10.3389\/fonc.2020.605380","article-title":"rategies for Targeting Gene Therapy in Cancer Cells with Tumor-Specific Promoters","volume":"10","year":"2020","journal-title":"Front. Oncol."},{"key":"ref_102","doi-asserted-by":"crossref","first-page":"149","DOI":"10.3389\/fonc.2017.00149","article-title":"Genomic signature of the natural oncolytic herpes simplex virus HF10 and its therapeutic role in preclinical and clinical trials","volume":"7","author":"Eissa","year":"2017","journal-title":"Front. Oncol."},{"key":"ref_103","doi-asserted-by":"crossref","unstructured":"Mostafa, A.A., Meyers, D.E., Thirukkumaran, C.M., Liu, P.J., Gratton, K., Spurrell, J., Shi, Q., Thakur, S., and Morris, D.G. (2018). Oncolytic Reovirus and immune checkpoint inhibitor as a novel immunotherapeutic strategy for breast cancer. Cancers, 10.","DOI":"10.3390\/cancers10060205"},{"key":"ref_104","doi-asserted-by":"crossref","first-page":"E4504","DOI":"10.1073\/pnas.1408759111","article-title":"Identification and characterization of alphavirus M1 as a selective oncolytic virus targeting ZAP-defective human cancers","volume":"111","author":"Lin","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_105","doi-asserted-by":"crossref","first-page":"7164","DOI":"10.1158\/1078-0432.CCR-11-0649","article-title":"A pilot study of MUC-1\/CEA\/TRICOM poxviral-based vaccine in patients with metastatic breast and ovarian cancer","volume":"17","author":"Mohebtash","year":"2011","journal-title":"Clin. Cancer Res."},{"key":"ref_106","doi-asserted-by":"crossref","first-page":"3328","DOI":"10.4161\/cc.8.20.9832","article-title":"Alphavirus M1 induces apoptosis of malignant glioma cells via downregulation and nucleolar translocation of p21WAF1\/CIP1 protein","volume":"8","author":"Hu","year":"2009","journal-title":"Cell Cycle"},{"key":"ref_107","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1089\/hum.2020.114","article-title":"Systematic Characterization of the Biodistribution of the Oncolytic Virus M1","volume":"31","author":"Cai","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"ref_108","doi-asserted-by":"crossref","first-page":"648","DOI":"10.1111\/j.1365-2990.2010.01110.x","article-title":"Semliki Forest virus-mediated gene therapy of the RG2 rat glioma","volume":"36","author":"Roche","year":"2010","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"ref_109","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1089\/hum.2014.100","article-title":"Intravenous administration of retroviral replicating vector, Toca 511, demonstrates efficacy in orthotopic immune-competent mouse glioma model","volume":"26","author":"Huang","year":"2015","journal-title":"Hum. Gene Ther."},{"key":"ref_110","unstructured":"(2023, January 11). Viral Therapy in Treating Patients with Recurrent Glioblastoma Multiforme, Available online: www.clinicaltrials.govNCT00390299."},{"key":"ref_111","doi-asserted-by":"crossref","first-page":"341ra75","DOI":"10.1126\/scitranslmed.aad9784","article-title":"Phase I trial of vocimagine amiroretrorepvec and 5-fluorocytosine for recurrent high-grade glioma","volume":"8","author":"Cloughesy","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"ref_112","unstructured":"(2022, January 11). Tocagen Reports Results of Toca 5 Phase 3 Trial in Recurrent Brain Cancer. Available online: https:\/\/bit.ly\/2lPm19v."},{"key":"ref_113","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1038\/gt.2008.169","article-title":"A Kunjin replicon vector encoding granulocyte macrophage colony-stimulating factor for intra-tumoral gene therapy","volume":"16","author":"Smeenk","year":"2009","journal-title":"Gene Ther."},{"key":"ref_114","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/j.jss.2019.07.032","article-title":"Immune effects of M51R vesicular stomatitis virus treatment of carcinomatosis from colon cancer","volume":"245","author":"Day","year":"2020","journal-title":"J. Surg. Res."},{"key":"ref_115","doi-asserted-by":"crossref","first-page":"779","DOI":"10.1021\/acs.molpharmaceut.8b01046","article-title":"Liposome Encapsulation of Oncolytic Virus M1 To Reduce Immunogenicity and Immune Clearance in Vivo","volume":"16","author":"Wang","year":"2019","journal-title":"Mol. Pharm."},{"key":"ref_116","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1038\/10548","article-title":"Cancer therapy using a self-replicating RNA vaccine","volume":"5","author":"Ying","year":"1999","journal-title":"Nat. Med."},{"key":"ref_117","doi-asserted-by":"crossref","first-page":"e001662","DOI":"10.1136\/jitc-2020-001662","article-title":"Long-term Survival of Patients with Stage III colon Cancer Treated with VRP-CEA(6D), an Alphavirus Vector that Increases the CD8+ Effector Memory T Cell to Treg Ratio","volume":"8","author":"Crosby","year":"2020","journal-title":"J. Immunother. Cancer"},{"key":"ref_118","doi-asserted-by":"crossref","first-page":"1492","DOI":"10.1038\/mt.2016.101","article-title":"Phase I study of intravenous oncolytic poxvirus (vvDD) in patients with advanced solid cancers","volume":"24","author":"Guo","year":"2016","journal-title":"Mol. Ther."},{"key":"ref_119","doi-asserted-by":"crossref","first-page":"607","DOI":"10.7785\/tcrt.2012.500414","article-title":"Recombinant Newcastle disease virus expressing IL15 demonstrates promising antitumor efficiency in melanoma model. Technol","volume":"14","author":"Niu","year":"2015","journal-title":"Cancer Res. Treat."},{"key":"ref_120","doi-asserted-by":"crossref","unstructured":"Kimpel, J., Urbiola, C., Koske, I., Tober, R., Banki, Z., Wollmann, G., and von Laer, D. (2018). The Oncolytic virus VSV-GP is effective against malignant melanoma. Viruses, 10.","DOI":"10.3390\/v10030108"},{"key":"ref_121","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1038\/gt.2009.161","article-title":"Single-cycle viral gene expression, rather than progressive replication and oncolysis, is required for VSV therapy of B16 melanoma","volume":"17","author":"Galivo","year":"2010","journal-title":"Gene Ther."},{"key":"ref_122","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1158\/1078-0432.CCR-0690-3","article-title":"Systemic therapy of malignant human melanoma tumors by a common cold-producing enterovirus, coxsackievirus a21","volume":"10","author":"Shafren","year":"2014","journal-title":"Clin. Cancer Res."},{"key":"ref_123","doi-asserted-by":"crossref","first-page":"611","DOI":"10.2217\/imt.15.35","article-title":"Talimogene laherparevec (T-VEC) for the treatment of advanced melanoma","volume":"7","author":"Johnson","year":"2015","journal-title":"Immunotherapy"},{"key":"ref_124","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1186\/s40425-016-0158-5","article-title":"Into the clinic: Talimigene laherparevec (T-VEC), a first-in-class intratumoral oncolytic viral therapy","volume":"4","author":"Rehman","year":"2016","journal-title":"J. Immunother. Ther. Cancer"},{"key":"ref_125","first-page":"3599","article-title":"A tumor-targeting adenovirus with high gene transduction efficiency for primary pancreatic cancer and ascites cells","volume":"37","author":"Nagasato","year":"2017","journal-title":"Anticancer Res."},{"key":"ref_126","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1586\/14760584.5.1.9","article-title":"Development of the panvac-vf vaccine for pancreatic cancer","volume":"5","author":"Petrulio","year":"2006","journal-title":"Expert Rev. Vaccines"},{"key":"ref_127","doi-asserted-by":"crossref","unstructured":"Hirooka, Y., Kasuya, H., Ishikawa, T., Hawashima, H., Ohno, E., Villalobos, I.B., Naoe, Y., Ichinose, T., Koyoma, N., and Goto, H. (2018). A phase I clinical trial of EUS-guided intratumoral injection of the oncolytic virus, HF10 for unresectable locally advanced pancreatic cancer. BMC Cancer, 18.","DOI":"10.1186\/s12885-018-4453-z"},{"key":"ref_128","doi-asserted-by":"crossref","first-page":"39152","DOI":"10.1021\/acsami.7b10796","article-title":"Ovarian cancer therapy by VSVMP gene mediated by a paclitaxel-enhanced nanoparticle","volume":"9","author":"Long","year":"2017","journal-title":"ACS Appl. Mater. Interfaces"},{"key":"ref_129","doi-asserted-by":"crossref","first-page":"1584","DOI":"10.1093\/annonc\/mdn167","article-title":"Efficient cisplatin-resistant human ovarian cancer growth and prolonged survival by gene transferred vesicular stomatitis virus matrix protein in nude mice","volume":"19","author":"Zhong","year":"2008","journal-title":"Ann. Oncol."},{"key":"ref_130","doi-asserted-by":"crossref","first-page":"4553","DOI":"10.1158\/1078-0432.CCR-04-2610","article-title":"Oncolytic Viral Therapy for Cervical and Ovarian Cancer Cells by Sindbis Virus AR339 Strain","volume":"11","author":"Unno","year":"2005","journal-title":"Clin. Cancer Res."},{"key":"ref_131","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1158\/0008-5472.CAN-09-2762","article-title":"Phase I trial of intraperitoneal administration of an oncolytic measles virus strain engineered to express carcinoembryonic antigen for recurrent ovarian cancer","volume":"70","author":"Galanis","year":"2010","journal-title":"Cancer Res."},{"key":"ref_132","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1002\/pros.20857","article-title":"Engineered measles virus as a novel oncolytic therapy against prostate cancer","volume":"69","author":"Msaouel","year":"2009","journal-title":"Prostate"},{"key":"ref_133","doi-asserted-by":"crossref","first-page":"1128","DOI":"10.1002\/pros.20962","article-title":"Prostate-specific membrane antigen retargeted measles virotherapy for the treatment of prostate cancer","volume":"69","author":"Liu","year":"2009","journal-title":"Prostate"},{"key":"ref_134","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1080\/07357907.2018.1434539","article-title":"Combination of vaccine-strain measles and mumps viruses enhances oncolytic activity against human solid malignancies","volume":"7","author":"Son","year":"2018","journal-title":"Cancer Investig."},{"key":"ref_135","doi-asserted-by":"crossref","first-page":"3999","DOI":"10.1158\/1078-0432.CCR-06-2202","article-title":"A Novel Alphavirus Vaccine Encoding Prostate-specific Membrane Antigen Elicits Potent Cellular and Humoral Immune Responses","volume":"13","author":"Durso","year":"2007","journal-title":"Clin. Cancer Res."},{"key":"ref_136","doi-asserted-by":"crossref","first-page":"1344","DOI":"10.1158\/0008-5472.CAN-06-2996","article-title":"In Vivo effects of Vaccination with Six-Transmembrane Epithelial Antigen of the Prostate: A Candidate Antigen for Treating Prostate Cancer","volume":"67","author":"Gray","year":"2007","journal-title":"Cancer Res."},{"key":"ref_137","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1158\/0008-5472.CAN-07-0445","article-title":"Prostate Stem Cell Antigen Vaccination Induces a Long-Term Protective Immune Response against Prostate Cancer in the Absence of Autoimmunity","volume":"68","author":"Gray","year":"2008","journal-title":"Cancer Res."},{"key":"ref_138","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1038\/gt.2016.5","article-title":"Oncolytic vaccinia virus as a vector for therapeutic sodium iodide symporter gene therapy in prostate cancer","volume":"23","author":"Mansfield","year":"2016","journal-title":"Gene Ther."},{"key":"ref_139","doi-asserted-by":"crossref","first-page":"943","DOI":"10.1016\/j.vaccine.2012.11.096","article-title":"A Phase I Dose Escalation Trial of Vaccine Replicon Particles (VRP) Expressing Prostate-specific Membrane Antigen (PSMA) in Subjects with Prostate Cancer","volume":"31","author":"Slovin","year":"2013","journal-title":"Vaccine"},{"key":"ref_140","first-page":"43","article-title":"Clinical testing of engineered oncolytic measles virus strains in the treatment of cancer: An overview","volume":"11","author":"Msaouel","year":"2009","journal-title":"Curr. Opin. Mol. Ther."},{"key":"ref_141","doi-asserted-by":"crossref","first-page":"540","DOI":"10.1016\/j.ygyno.2006.09.022","article-title":"Efficient inhibition of intraperitoneal human ovarian cancer growth and prolonged survival by gene transfer of vesicular stomatitis virus matrix protein in nude mice","volume":"104","author":"Lin","year":"2007","journal-title":"Gynecol. Oncol."},{"key":"ref_142","doi-asserted-by":"crossref","first-page":"793","DOI":"10.1073\/pnas.97.2.793","article-title":"Adenoviral gene transfer of SERCa2a improves left-ventricular function in aortic-banded rats in transition to heart failure","volume":"97","author":"Miyamoto","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_143","doi-asserted-by":"crossref","first-page":"H1204","DOI":"10.1152\/ajpheart.00892.2006","article-title":"Transcoronary transfer of SERCa2a increases coronary blood flow and decreases cardiomyocyte size in a type 2 diabetic rat model","volume":"292","author":"Sakata","year":"2007","journal-title":"Am. J. Physiol. Heart Circ. Physiol."},{"key":"ref_144","doi-asserted-by":"crossref","first-page":"1284","DOI":"10.1038\/mt.2010.77","article-title":"SERCA2a gene transfer enhances eNOS expression and activity in endothelial cells","volume":"18","author":"Hadri","year":"2010","journal-title":"Mol. Ther."},{"key":"ref_145","doi-asserted-by":"crossref","first-page":"1026","DOI":"10.1038\/mt.2008.61","article-title":"Lentiviral vector\u2013mediated SERCA2 gene transfer protects against heart failure and left ventricular remodeling after myocardial infarction in rats","volume":"16","author":"Niwano","year":"2008","journal-title":"Mol. Ther."},{"key":"ref_146","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1007\/s11033-009-9665-5","article-title":"Improvement of heart function in postinfarct heart failure swine models after hepatocyte growth factor transfer: Comparison of low-, medium- and high-dose groups","volume":"37","author":"Yang","year":"2010","journal-title":"Mol. Biol. Rep."},{"key":"ref_147","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1161\/CIRCULATIONAHA.111.053272","article-title":"Connexin gene transfer preserves conduction velocity and prevents atrial fibrillation","volume":"125","author":"Igarashi","year":"2012","journal-title":"Circulation"},{"key":"ref_148","doi-asserted-by":"crossref","first-page":"2263","DOI":"10.1161\/CIRCULATIONAHA.109.911156","article-title":"Selective molecular potassium channel blockade prevents atrial fibrillation","volume":"121","author":"Amit","year":"2010","journal-title":"Circulation"},{"key":"ref_149","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1038\/nature11044","article-title":"In vivo reprogramming of murine cardiac fibroblasts into induced cardiomyocytes","volume":"485","author":"Qian","year":"2012","journal-title":"Nature"},{"key":"ref_150","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1038\/s41434-021-00295-1","article-title":"Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-D\u0394N\u0394C gene therapy eight-year follow-up of phase I KAT301 study","volume":"29","author":"Leikas","year":"2022","journal-title":"Gene Ther."},{"key":"ref_151","doi-asserted-by":"crossref","first-page":"1503","DOI":"10.1038\/sj.gt.3302802","article-title":"Angiogenic gene therapy in patients with nonrevascularizable ischemic heart disease: A phase 2 randomized, controlled trial of AdVEGF(121) (AdVEGF121) versus maximum medical treatment","volume":"13","author":"Stewart","year":"2006","journal-title":"Gene Ther."},{"key":"ref_152","doi-asserted-by":"crossref","first-page":"1291","DOI":"10.1161\/hc1102.105595","article-title":"Angiogenic Gene Therapy (AGENT) trial in patients with stable angina pectoris","volume":"105","author":"Grines","year":"2002","journal-title":"Circulation"},{"key":"ref_153","doi-asserted-by":"crossref","first-page":"1038","DOI":"10.1016\/j.jacc.2007.06.010","article-title":"Effects of Ad5FGF-4 in patients with angina: An analysis of pooled data from the AGENT-3 and AGENT-4 trials","volume":"50","author":"Henry","year":"2007","journal-title":"J. Am. Coll. Cardiol."},{"key":"ref_154","doi-asserted-by":"crossref","first-page":"1339","DOI":"10.1016\/S0735-1097(03)00988-4","article-title":"A randomized, double-blind, placebo-controlled trial of Ad5FGF-4 gene therapy and its effect on myocardial perfusion in patients with stable angina","volume":"42","author":"Grines","year":"2003","journal-title":"J. Am. Coll. Cardiol."},{"key":"ref_155","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.cardfail.2009.01.013","article-title":"Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease (CUPID) Trial Investigators. Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1\/2 clinical trial","volume":"15","author":"Jaski","year":"2009","journal-title":"J. Card. Fail."},{"key":"ref_156","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1161\/CIRCULATIONAHA.111.022889","article-title":"Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) Investigators. Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): A phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure","volume":"124","author":"Jessup","year":"2011","journal-title":"Circulation"},{"key":"ref_157","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1161\/CIRCRESAHA.113.302421","article-title":"Long-term effects of AAV1\/SERCA2a gene transfer in patients with severe heart failure: Analysis of recurrent cardiovascular events and mortality","volume":"114","author":"Zsebo","year":"2014","journal-title":"Circ. Res."},{"key":"ref_158","doi-asserted-by":"crossref","first-page":"1642","DOI":"10.1038\/sj.gt.3300775","article-title":"Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus","volume":"5","author":"Watson","year":"1998","journal-title":"Gene Ther."},{"key":"ref_159","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1002\/jgm.554","article-title":"Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia","volume":"6","author":"Lebherz","year":"2004","journal-title":"J. Gene Med."},{"key":"ref_160","doi-asserted-by":"crossref","first-page":"e8791","DOI":"10.15252\/emmm.201708791","article-title":"FGF21 gene therapy as treatment for obesity and insulin resistance","volume":"10","author":"Jimenez","year":"2018","journal-title":"EMBO Mol. Med."},{"key":"ref_161","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1016\/j.omtm.2019.12.014","article-title":"Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase","volume":"19","author":"Tao","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_162","doi-asserted-by":"crossref","first-page":"eeam6375","DOI":"10.1126\/scitranslmed.aam6375","article-title":"Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase","volume":"9","author":"Puzzo","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"ref_163","doi-asserted-by":"crossref","first-page":"1872","DOI":"10.1007\/s00268-007-9168-2","article-title":"Reversal of diabetes in mice by intrahepatic injection of bone-derived GFP-murine mesenchymal stem cells infected with the recombinant retrovirus-carrying human insulin gene","volume":"31","author":"Xu","year":"2007","journal-title":"World J. Surg."},{"key":"ref_164","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1016\/j.biochi.2015.12.002","article-title":"An adenovirus-derived protein: A novel candidate for anti-diabetic drug development","volume":"121","author":"Hegde","year":"2016","journal-title":"Biochemie"},{"key":"ref_165","doi-asserted-by":"crossref","first-page":"776","DOI":"10.1016\/j.jhep.2016.05.012","article-title":"Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria","volume":"65","author":"Sangro","year":"2016","journal-title":"J. Hepatol."},{"key":"ref_166","doi-asserted-by":"crossref","first-page":"16363","DOI":"10.1073\/pnas.0904514106","article-title":"Sustained transgene expression despite Tlymphocyte responses in a clinical trial of rAAV1-AAT gene therapy","volume":"106","author":"Brantly","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_167","doi-asserted-by":"crossref","first-page":"1239","DOI":"10.1089\/hum.2011.053","article-title":"Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alphal-antitrypsin: Interim results","volume":"22","author":"Flotte","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"ref_168","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1182\/blood.V95.3.820.003k32_820_828","article-title":"Sustained high-level expression of full-length human factor VIII and a restoration of clotting activity in hemophilic mice using a minimal adenovirus vector","volume":"95","author":"Zhou","year":"2000","journal-title":"Blood"},{"key":"ref_169","doi-asserted-by":"crossref","first-page":"1401","DOI":"10.1073\/pnas.92.5.1401","article-title":"Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows long-term expression","volume":"92","author":"Dai","year":"1995","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_170","doi-asserted-by":"crossref","first-page":"2353","DOI":"10.1073\/pnas.91.6.2353","article-title":"In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs","volume":"91","author":"Kay","year":"1994","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_171","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.1089\/hum.1995.6.8-1039","article-title":"Gene therapy for hemophilia B: Host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression","volume":"6","author":"Fang","year":"1995","journal-title":"Hum. Gene Ther."},{"key":"ref_172","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1182\/blood-2005-12-5115","article-title":"Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs","volume":"108","author":"Jiang","year":"2006","journal-title":"Blood"},{"key":"ref_173","doi-asserted-by":"crossref","unstructured":"Callan, M.B., Haskins, M.E., Wang, P., Zhou, S., High, K.A., and Arruda, V.R. (2016). Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs. PLoS ONE, 11.","DOI":"10.1371\/journal.pone.0151800"},{"key":"ref_174","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/s41587-020-0741-7","article-title":"A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells","volume":"39","author":"Nguyen","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"ref_175","doi-asserted-by":"crossref","first-page":"1553","DOI":"10.1182\/blood-2014-07-588194","article-title":"AAV liver expression of FIXPadua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice","volume":"125","author":"Crudele","year":"2015","journal-title":"Blood"},{"key":"ref_176","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1182\/hematology.2019000007","article-title":"Gene therapy for hemophilia","volume":"2019","author":"Nathwani","year":"2019","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program"},{"key":"ref_177","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1056\/NEJMoa1908490","article-title":"Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A","volume":"382","author":"Pasi","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"ref_178","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","article-title":"Long-term safety and efficacy of factor IX gene therapy in hemophilia B","volume":"371","author":"Nathwani","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"ref_179","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1056\/NEJMoa2119913","article-title":"Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B","volume":"387","author":"Chowdary","year":"2022","journal-title":"N. Engl. J. Med."},{"key":"ref_180","doi-asserted-by":"crossref","first-page":"3335","DOI":"10.1016\/j.ymthe.2022.09.020","article-title":"First conditional marketing authorization approval in the European Union for hemophilia \u201cA\u201d gene therapy","volume":"30","author":"VandenDriessche","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_181","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1111\/j.1538-7836.2007.02346.x","article-title":"Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A","volume":"5","author":"Shi","year":"2007","journal-title":"J. Tromb. Haemost."},{"key":"ref_182","doi-asserted-by":"crossref","first-page":"277ra28","DOI":"10.1126\/scitranslmed.aaa1405","article-title":"Liver-directed lentiviral gene therapy in a dog model of hemophilia B","volume":"7","author":"Cantore","year":"2015","journal-title":"Sci. Transl. Med."},{"key":"ref_183","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1002\/jcp.29861","article-title":"In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy","volume":"236","author":"Chen","year":"2021","journal-title":"J. Cell Physiol."},{"key":"ref_184","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1038\/mt.2016.87","article-title":"Safe and efficient gene therapy for pyruvate kinase deficiency","volume":"24","author":"Calabria","year":"2016","journal-title":"Mol. Ther."},{"key":"ref_185","doi-asserted-by":"crossref","first-page":"211","DOI":"10.1038\/sj.mt.6300033","article-title":"Stem Cell Collection and Gene Transfer in Fanconi Anemia","volume":"15","author":"Kelly","year":"2007","journal-title":"Mol. Ther."},{"key":"ref_186","doi-asserted-by":"crossref","first-page":"1792","DOI":"10.3324\/haematol.2014.111195","article-title":"Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-defificent Diamond-Blackfan anemia","volume":"99","author":"Jaako","year":"2014","journal-title":"Haematologica"},{"key":"ref_187","doi-asserted-by":"crossref","first-page":"1106","DOI":"10.1089\/hum.2018.122","article-title":"Gene Therapy for Hemoglobinopathies","volume":"29","author":"Cavazzana","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_188","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1182\/blood-2019-128482","article-title":"Northstar-3: Interim results from a phase 3 study evaluating lentiglobin gene therapy in patients with transfusion-dependent \u03b2-thalassemia and either a \u03b20 or IVS-I-110 mutation at both alleles of the HBB gene","volume":"134","author":"Lal","year":"2019","journal-title":"Blood"},{"key":"ref_189","doi-asserted-by":"crossref","first-page":"11592","DOI":"10.1038\/s41598-019-48078-4","article-title":"In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques","volume":"9","author":"Shangaris","year":"2019","journal-title":"Sci. Rep."},{"key":"ref_190","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1038\/s41591-018-0301-6","article-title":"Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ss-thalassemia","volume":"25","author":"Marktel","year":"2019","journal-title":"Nat. Med."},{"key":"ref_191","doi-asserted-by":"crossref","first-page":"2368","DOI":"10.1126\/science.1065806","article-title":"Correction of sickle cell disease in transgenic mouse models by gene therapy","volume":"294","author":"Pawliuk","year":"2001","journal-title":"Science"},{"key":"ref_192","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1056\/NEJMoa1609677","article-title":"Gene therapy in a patient with sickle cell disease","volume":"376","author":"Ribeil","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"ref_193","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1089\/hum.2018.061","article-title":"Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study","volume":"29","author":"Urbinati","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_194","doi-asserted-by":"crossref","first-page":"1662","DOI":"10.1038\/sj.gt.3303024","article-title":"HIV vector-mediated targeted suicide gene therapy for adult T-cell leukemia","volume":"14","author":"Miyake","year":"2007","journal-title":"Gene Ther."},{"key":"ref_195","doi-asserted-by":"crossref","first-page":"350","DOI":"10.4161\/cbt.9.5.10879","article-title":"Gene therapy for acute myeloid leukemia using Sindbis vectors expressing a fusogenic membrane glycoprotein","volume":"9","author":"Tan","year":"2010","journal-title":"Cancer Biol. Ther."},{"key":"ref_196","doi-asserted-by":"crossref","first-page":"2404","DOI":"10.1021\/acs.bioconjchem.9b00511","article-title":"A CD33 Antigen-Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Meyloid Leukemia","volume":"30","author":"Khan","year":"2019","journal-title":"Bioconjug. Chem."},{"key":"ref_197","doi-asserted-by":"crossref","first-page":"2851","DOI":"10.1007\/s00262-021-02895-7","article-title":"Boosting CAR T-cell responses in lymphoma by simultaneous targeting of CD40\/4-1BB using oncolytic viral gene therapy","volume":"70","author":"Wenthe","year":"2021","journal-title":"Cancer Immunol. Immunother."},{"key":"ref_198","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1182\/blood.V93.2.643.402k24_643_654","article-title":"Eradication of pre-established lymphoma using herpes simplex virus amplicon vectors","volume":"93","author":"Kutubuddin","year":"1999","journal-title":"Blood"},{"key":"ref_199","doi-asserted-by":"crossref","first-page":"762","DOI":"10.1016\/j.ymthe.2020.09.041","article-title":"Oncolytic Virus Therapy with HSV-1 for Hematological Malignancies","volume":"29","author":"Ishino","year":"2021","journal-title":"Mol. Ther."},{"key":"ref_200","doi-asserted-by":"crossref","unstructured":"Han, T., Abdel-Motal, U.M., Chang, D.-K., Sui, J., Muvaffak, A., Campbell, J., Zhu, Q., Kupper, T.S., and Marasco, W.A. (2012). Human anti-CCR4 minibody gene transfer for the treatment of cutaneous T-cell lymphoma. PLoS ONE, 7.","DOI":"10.1371\/journal.pone.0044455"},{"key":"ref_201","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1186\/s40425-019-0664-3","article-title":"Molecular and metabolic pathways mediating curative treatment of a non-Hodgkin B cell lymphoma by Sindbis viral vectors and anti-4-1BB monoclonal antibody","volume":"7","author":"Yu","year":"2019","journal-title":"J. Immunother. Cancer"},{"key":"ref_202","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1038\/mt.2011.68","article-title":"Myeloma xenograft destruction by a nonviral vector delivering oncolytic infectious nucleic acid","volume":"19","author":"Hadac","year":"2011","journal-title":"Mol. Ther."},{"key":"ref_203","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1038\/cgt.2012.14","article-title":"Potent systemic therapy of multiple myeloma utilizing oncolytic vesicular stomatitis virus coding for interferon-\u03b2","volume":"19","author":"Naik","year":"2012","journal-title":"Cancer Gene Ther."},{"key":"ref_204","doi-asserted-by":"crossref","first-page":"3023","DOI":"10.1038\/onc.2011.478","article-title":"Reovirus therapy stimulated endoplasmic reticular stress, NOXA induction, and augments bortezomib-mediated apoptosis in multiple myeloma","volume":"31","author":"Kelly","year":"2012","journal-title":"Oncogene"},{"key":"ref_205","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1038\/gt.2008.64","article-title":"Potential of AAV vectors in the treatment of metabolic disease","volume":"15","author":"Alexander","year":"2008","journal-title":"Gene Ther."},{"key":"ref_206","doi-asserted-by":"crossref","first-page":"15","DOI":"10.3233\/JND-190426","article-title":"Advancements in AAV-mediated Gene Therapy for Pompe Disease","volume":"7","author":"Salabarria","year":"2020","journal-title":"J. Neuromusc. Dis."},{"key":"ref_207","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1055\/s-0037-1613761","article-title":"Structural basis for hemophilia A caused by mutations in the C domains of blood coagulation factor VIII","volume":"83","author":"Gale","year":"2000","journal-title":"Thromb. Haemost."},{"key":"ref_208","doi-asserted-by":"crossref","first-page":"1225","DOI":"10.1182\/blood.V79.5.1225.1225","article-title":"Hemophilia B caused by five different nondeletion mutations in the protease domain of factor IX","volume":"79","author":"Ludwig","year":"1992","journal-title":"Blood"},{"key":"ref_209","doi-asserted-by":"crossref","first-page":"349","DOI":"10.2174\/1566523054065048","article-title":"AAV-mediated gene transfer for treatment of hemophilia","volume":"5","author":"Wang","year":"2005","journal-title":"Curr. Gene Ther."},{"key":"ref_210","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1046\/j.1538-7836.2003.00096.x","article-title":"A single adenoassociated virus (AAV)-murine factor FVIII","volume":"1","author":"Sarkar","year":"2003","journal-title":"J. Thromb. Haemost."},{"key":"ref_211","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1182\/blood-2003-08-2954","article-title":"Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype","volume":"103","author":"Sarkar","year":"2004","journal-title":"Blood"},{"key":"ref_212","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1517\/14712598.2012.665047","article-title":"Alternative options for DNA-based experimental therapy of \u03b2-thalassemia","volume":"12","author":"Gambari","year":"2012","journal-title":"Expert Opin. Biol. Ther."},{"key":"ref_213","doi-asserted-by":"crossref","first-page":"792","DOI":"10.1038\/178792a0","article-title":"A specific chemical difference between the globins of normal human and sickle cell anemia hemoglobin","volume":"178","author":"Ingram","year":"1956","journal-title":"Nature"},{"key":"ref_214","unstructured":"Lundstrom, K. (2023). Comprehensive Hematology and Stem Cell Research, Elsevier. in press."},{"key":"ref_215","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1007\/978-3-211-78205-7_17","article-title":"AAV-GAD gene for rat models of neuropathic pain and Parkinson\u2019s disease","volume":"101","author":"Kim","year":"2008","journal-title":"Acta Neurochir. Suppl."},{"key":"ref_216","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1016\/S0006-8993(00)02915-2","article-title":"Towards a neuroprotective gene therapy for Parkinson\u2019s disease: Use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat Parkinson model","volume":"886","author":"Kirik","year":"2000","journal-title":"Brain Res."},{"key":"ref_217","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1126\/science.290.5492.767","article-title":"Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate model of Parkinson\u2019s disease","volume":"290","author":"Kordower","year":"2000","journal-title":"Science"},{"key":"ref_218","doi-asserted-by":"crossref","first-page":"1980","DOI":"10.1212\/01.wnl.0000312381.29287.ff","article-title":"Results from a phase I safety trial of hAADC gene therapy for Parkinson disease","volume":"70","author":"Eberling","year":"2008","journal-title":"Neurology"},{"key":"ref_219","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1016\/S0140-6736(13)61939-X","article-title":"Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson\u2019s disease: A dose escalation, open-label, phase 1\/2 trial","volume":"383","author":"Palfi","year":"2014","journal-title":"Lancet"},{"key":"ref_220","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1089\/humc.2018.081","article-title":"Long-Term Follow-up of a Phase I\/II of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson\u2019s Disease","volume":"29","author":"Palfi","year":"2018","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"ref_221","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/j.brainres.2005.08.056","article-title":"AAV2\/5-mediated NGF gene delivery protects septal cholinergic neurons following axotomy","volume":"1061","author":"Wu","year":"2005","journal-title":"Brain Res."},{"key":"ref_222","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1007\/s00401-015-1498-9","article-title":"Viral gene transfer of APPs\u03b1 rescues synaptic failure in an Alzheimer\u2019s disease model","volume":"131","author":"Fol","year":"2016","journal-title":"Acta Neuropathol."},{"key":"ref_223","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1001\/jamaneurol.2018.0233","article-title":"Adeno-associated viral vector (Serotype 2)\u2013nerve growth factor for patients with Alzheimer Disease","volume":"75","author":"Rafii","year":"2018","journal-title":"JAMA Neurol."},{"key":"ref_224","doi-asserted-by":"crossref","first-page":"961","DOI":"10.1111\/cns.12312","article-title":"Lenti-GDNF gene therapy protects against Alzheimer\u2019s disease-like neuropathology in 3xTg-AD mice and MC65 cells","volume":"20","author":"Revilla","year":"2014","journal-title":"CNS Neurosci. Ther."},{"key":"ref_225","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1016\/j.neurobiolaging.2019.02.003","article-title":"Lentiviral vector-mediated overexpression of klotho in the brain improves Alzheimer\u2019s disease-like pathology and cognitive deficits in mice","volume":"78","author":"Zeng","year":"2019","journal-title":"Neurobiol. Aging"},{"key":"ref_226","doi-asserted-by":"crossref","first-page":"630","DOI":"10.1038\/gt.2017.71","article-title":"AAV5-miHTT gene therapy demonstrates suppression of huntingtin aggregation and neuronal dysfunction in a rat model of Huntington\u2019s disease","volume":"24","author":"Miniarikova","year":"2017","journal-title":"Gene Ther."},{"key":"ref_227","doi-asserted-by":"crossref","first-page":"2163","DOI":"10.1016\/j.ymthe.2018.06.021","article-title":"AAV5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in Huntington\u2019s disease minipig model","volume":"26","author":"Evers","year":"2018","journal-title":"Mol. Ther."},{"key":"ref_228","doi-asserted-by":"crossref","first-page":"185","DOI":"10.3233\/JHD-200002","article-title":"Huntington\u2019s Disease Clinical Trials Corner: April 2020","volume":"9","author":"Rodrigues","year":"2020","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_229","first-page":"153","article-title":"Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome","volume":"24","author":"Sinnett","year":"2017","journal-title":"Discov. Med."},{"key":"ref_230","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1038\/s41434-019-0085-4","article-title":"AAV9 vector: A novel modality in gene therapy for spinal muscular atrophy","volume":"26","author":"Pattali","year":"2019","journal-title":"Gene Ther."},{"key":"ref_231","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-dose gene-replacement therapy for spinal muscular atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"ref_232","doi-asserted-by":"crossref","first-page":"1255","DOI":"10.1007\/s40265-019-01162-5","article-title":"Onasemnogene Abeparvovec First Global Approval","volume":"79","author":"Hoy","year":"2019","journal-title":"Drugs"},{"key":"ref_233","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/j.ymthe.2017.02.019","article-title":"Progress toward gene therapy for Duchenne muscular dystrophy","volume":"25","author":"Chamberlain","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_234","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1016\/S0014-5793(98)00251-8","article-title":"Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs","volume":"425","author":"Yuasa","year":"1998","journal-title":"FEBS Lett."},{"key":"ref_235","doi-asserted-by":"crossref","first-page":"1265","DOI":"10.1016\/S0006-291X(02)00362-5","article-title":"Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene","volume":"293","author":"Sakamoto","year":"2002","journal-title":"Biochem. Biophys. Res. Comm."},{"key":"ref_236","doi-asserted-by":"crossref","first-page":"787","DOI":"10.1038\/nm1439","article-title":"rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice","volume":"12","author":"Gregorevic","year":"2006","journal-title":"Nat. Med."},{"key":"ref_237","doi-asserted-by":"crossref","first-page":"1501","DOI":"10.1038\/mt.2012.111","article-title":"Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: A preclinical model for human therapies","volume":"20","author":"Wang","year":"2012","journal-title":"Mol. Ther."},{"key":"ref_238","doi-asserted-by":"crossref","first-page":"1122","DOI":"10.1001\/jamaneurol.2020.1484","article-title":"Assessment of systemic delivery of rAAVrh74. MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy","volume":"77","author":"Mendell","year":"2020","journal-title":"JAMA Neurol."},{"key":"ref_239","doi-asserted-by":"crossref","first-page":"14848","DOI":"10.1038\/ncomms14848","article-title":"PABPN1 gene therapy for oculopharyngeal muscular dystrophy","volume":"8","author":"Malerba","year":"2017","journal-title":"Nat. Commun."},{"key":"ref_240","first-page":"35ra42","article-title":"temic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy","volume":"235","author":"Valori","year":"2010","journal-title":"Sci. Transl. Med."},{"key":"ref_241","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1002\/ana.24332","article-title":"A large animal model of spinal muscular atrophy and correction of phenotype","volume":"77","author":"Duque","year":"2015","journal-title":"Ann. Neurol."},{"key":"ref_242","first-page":"669","article-title":"Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease","volume":"28","author":"Gross","year":"2000","journal-title":"Science"},{"key":"ref_243","doi-asserted-by":"crossref","first-page":"e20190607","DOI":"10.1084\/jem.20190607","article-title":"Gene therapy for severe combined immunodeficiencies and beyond","volume":"217","author":"Fischer","year":"2020","journal-title":"J. Exp. Med."},{"key":"ref_244","doi-asserted-by":"crossref","first-page":"2181","DOI":"10.1016\/S0140-6736(04)17590-9","article-title":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector","volume":"364","author":"Gaspar","year":"2004","journal-title":"Lancet"},{"key":"ref_245","doi-asserted-by":"crossref","first-page":"3143","DOI":"10.1172\/JCI35798","article-title":"Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients","volume":"118","author":"Howe","year":"2008","journal-title":"J. Clin. Investig."},{"key":"ref_246","doi-asserted-by":"crossref","first-page":"852","DOI":"10.1016\/j.jaci.2018.08.024","article-title":"Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency","volume":"143","author":"Kohn","year":"2019","journal-title":"J. Allergy Clin. Immunol."},{"key":"ref_247","unstructured":"Butterfield, R., Shieh, P., Geffen, D., Yong, F., Binks, M., McDonnell, T.G., Ryan, K.A., Belluscio, B., Neelakanten, S., and Levy, D. (2023, January 11). One Year Data from Ambulatory Boys in a Phase 1b, Open-Label Study of Fordadistrogene Movaparvovec (PF-06939926) for Duchenne Muscular Dystrophy (DMD). Available online: www.mdaconference.org\/abstract-library\/one-year-data-from-ambulatory-boys-in-a-phase-1b-open-label-study-of-fordadistrogene-movaparvovec-pf-06939926-for-duchenne-muscular-dystrophy-dmd\/."},{"key":"ref_248","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"ref_249","doi-asserted-by":"crossref","first-page":"2002","DOI":"10.1056\/NEJMoa2027675","article-title":"Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency","volume":"384","author":"Kohn","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_250","doi-asserted-by":"crossref","first-page":"111","DOI":"10.2174\/1566523222666220921112753","article-title":"Gene Therapy Cargoes Based on Viral Vector Delivery","volume":"23","author":"Lundstrom","year":"2023","journal-title":"Curr Gene Ther."},{"key":"ref_251","doi-asserted-by":"crossref","first-page":"4357","DOI":"10.1167\/iovs.02-1332","article-title":"Gene therapy with brain-derived neurotrophic factor as a protection: Retinal ganglion cells in a rat glaucoma model","volume":"44","author":"Martin","year":"2003","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"ref_252","doi-asserted-by":"crossref","first-page":"1230","DOI":"10.1093\/hmg\/ddx028","article-title":"Therapeutic potential of AAV-mediated MMP-3 secretion from corneal endothelium in treating glaucoma","volume":"26","author":"Crosbie","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"ref_253","doi-asserted-by":"crossref","first-page":"376","DOI":"10.1089\/hum.2015.142","article-title":"Preclinical dose-escalation study of intravitreal AAV-RS1 gene therapy in a mouse model of X-linked retinoschisis: Dose-dependent expression and improved retinal structure and function","volume":"27","author":"Bush","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"ref_254","first-page":"543","article-title":"Gene Therapy for Color Blindness","volume":"90","author":"Hassall","year":"2017","journal-title":"Yale J. Biol. Med."},{"key":"ref_255","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1038\/nm1596","article-title":"Restoration of cone vision in a mouse model of achromatopsia","volume":"13","author":"Alexander","year":"2007","journal-title":"Nat. Med."},{"key":"ref_256","doi-asserted-by":"crossref","first-page":"2581","DOI":"10.1093\/hmg\/ddq136","article-title":"Gene therapy rescues cone function in congenital achromatopsia","volume":"19","author":"Alexander","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"ref_257","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/S0140-6736(17)30979-0","article-title":"Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: A phase I, open-label trial","volume":"390","author":"Heier","year":"2017","journal-title":"Lancet"},{"key":"ref_258","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1016\/j.ebiom.2016.11.016","article-title":"Phase 2a randomized clinical trial: Safety and post hoc analysis of subretinal rAAV.sFLT-1 for wet age-related macular degeneration","volume":"14","author":"Constable","year":"2016","journal-title":"EBioMedicine"},{"key":"ref_259","doi-asserted-by":"crossref","first-page":"1621","DOI":"10.1016\/j.ophtha.2017.05.016","article-title":"Gene therapy for Leder hereditary optic neuropathy: Low and medium-dose visual results","volume":"124","author":"Guy","year":"2017","journal-title":"Ophthalmology"},{"key":"ref_260","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1016\/j.ophtha.2017.12.036","article-title":"Safety of rAAV2\/2-ND4 gene therapy","volume":"125","author":"Vignal","year":"2018","journal-title":"Ophthalmology"},{"key":"ref_261","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: A randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"ref_262","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.ymthe.2020.11.029","article-title":"Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy","volume":"29","author":"Maguire","year":"2021","journal-title":"Mol. Ther."},{"key":"ref_263","doi-asserted-by":"crossref","first-page":"10613","DOI":"10.1073\/pnas.90.22.10613","article-title":"Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector","volume":"90","author":"Flotte","year":"1993","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_264","first-page":"658","article-title":"Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung","volume":"3","author":"Conrad","year":"1996","journal-title":"Gene Ther."},{"key":"ref_265","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1089\/hum.2017.201","article-title":"Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy","volume":"29","author":"Cao","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_266","doi-asserted-by":"crossref","first-page":"1961","DOI":"10.1089\/10430340260355365","article-title":"Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer","volume":"13","author":"Limberis","year":"2002","journal-title":"Hum. Gene Ther."},{"key":"ref_267","doi-asserted-by":"crossref","first-page":"e88730","DOI":"10.1172\/jci.insight.88730","article-title":"Lentiviral-mediated phenotypic correction of cystic fibrosis pigs","volume":"1","author":"Cooney","year":"2016","journal-title":"JCI Insight"},{"key":"ref_268","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1136\/thoraxjnl-2016-208406","article-title":"Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis","volume":"72","author":"Alton","year":"2017","journal-title":"Thorax"},{"key":"ref_269","doi-asserted-by":"crossref","first-page":"1979","DOI":"10.1016\/S0140-6736(20)32466-1","article-title":"Safety and immunogenicity of ChAdOx1 nCov-19 vaccine adminsitered in a prime-boost regimen in young and old adults (COV002): A singe-blind, randomised, controlled phase 2\/3 trial","volume":"336","author":"Ramasamy","year":"2020","journal-title":"Lancet"},{"key":"ref_270","doi-asserted-by":"crossref","unstructured":"Lundstrom, K. (2021). Viral Vectors for COVID-19 Vaccine Development. Viruses, 13.","DOI":"10.3390\/v13020317"},{"key":"ref_271","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/S0140-6736(20)31605-6","article-title":"Immunogenicity and safety of a recombinant adenovirus type-5-vectored COVID-19 vaccine in healthy adults aged 18 years or older: A randomised, double-blind, placebo-controlled, phase 2 trial","volume":"396","author":"Zhu","year":"2020","journal-title":"Lancet"},{"key":"ref_272","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/S0140-6736(21)00234-8","article-title":"Safety and efficacy of an rAd26 and rAd5 vector-based heterologous prime-boost COVID-19: An interim analysis of a randomised controlled phase 3 in Russia","volume":"397","author":"Logunov","year":"2021","journal-title":"Lancet"},{"key":"ref_273","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1038\/d41586-020-02386-2","article-title":"Russia\u2019s fast-track coronavirus vaccine draws outrage over safety","volume":"584","author":"Callaway","year":"2020","journal-title":"Nature"},{"key":"ref_274","doi-asserted-by":"crossref","first-page":"1535","DOI":"10.1001\/jama.2021.3645","article-title":"Immunogenicity of the Ad26.COV2.S vaccine for COVID-19","volume":"325","author":"Stephenson","year":"2021","journal-title":"JAMA"},{"key":"ref_275","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1016\/S0140-6736(15)61117-5","article-title":"Efficacy and effectiveness of an rVSV-vectored vaccine expressing Ebola surface glycoprotein: Interim results from the Guinea ring vaccination cluster-randomised trial","volume":"386","author":"Longini","year":"2015","journal-title":"Lancet"},{"key":"ref_276","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/S0140-6736(16)32621-6","article-title":"Efficacy and effectiveness of an rVSV-vectored vaccine in preventing Ebola virus disease: Final results from the Guinea ring vaccination, open-label, cluster-randomised trial (Ebola Ca Suffit!)","volume":"389","author":"Camacho","year":"2017","journal-title":"Lancet"},{"key":"ref_277","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1016\/j.cell.2020.03.011","article-title":"A vaccine against Ebola virus","volume":"181","year":"2020","journal-title":"Cell"},{"key":"ref_278","first-page":"2016","article-title":"Efficacy and safety of rAAV2-ND4 treatment for Leber\u2019s hereditary optic neuropathy","volume":"6","author":"Wan","year":"2016","journal-title":"Sci. Rep."},{"key":"ref_279","doi-asserted-by":"crossref","first-page":"1891","DOI":"10.1016\/S0140-6736(09)60327-5","article-title":"Cystic fibrosis","volume":"373","author":"Freedman","year":"2009","journal-title":"Lancet"},{"key":"ref_280","doi-asserted-by":"crossref","first-page":"13","DOI":"10.2174\/1874467210801010013","article-title":"Gene therapy: The first approved gene-based medicines, molecular mechanisms and clinical indications","volume":"1","author":"Pikkarainen","year":"2008","journal-title":"Curr. Mol. Pharmacol."},{"key":"ref_281","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1089\/hum.2017.218","article-title":"The first approved gene therapy product for cancer ad-p53 (Gendicine): 12 years in the clinic","volume":"29","author":"Zhang","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_282","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/mt.2014.248","article-title":"Glybera\u2019s second act: The curtain rises on the high cost of therapy","volume":"23","year":"2015","journal-title":"Mol. Ther."}],"container-title":["Viruses"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1999-4915\/15\/3\/698\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T18:50:21Z","timestamp":1760122221000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1999-4915\/15\/3\/698"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,3,7]]},"references-count":282,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2023,3]]}},"alternative-id":["v15030698"],"URL":"https:\/\/doi.org\/10.3390\/v15030698","relation":{},"ISSN":["1999-4915"],"issn-type":[{"value":"1999-4915","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,3,7]]}}}