{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T21:56:53Z","timestamp":1793051813835,"version":"4.5.0"},"reference-count":81,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2024,6,1]],"date-time":"2024-06-01T00:00:00Z","timestamp":1717200000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,6,1]],"date-time":"2024-06-01T00:00:00Z","timestamp":1717200000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T00:00:00Z","timestamp":1714521600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004336","name":"Novartis AG","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004336","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2024,6]]},"DOI":"10.1016\/j.omtm.2024.101263","type":"journal-article","created":{"date-parts":[[2024,5,4]],"date-time":"2024-05-04T09:50:02Z","timestamp":1714816202000},"page":"101263","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":19,"title":["Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus"],"prefix":"10.1016","volume":"32","author":[{"given":"Beltran","family":"Borges","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonia","family":"Varthaliti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marisa","family":"Schwab","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria T.","family":"Clarke","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Pivetti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nalin","family":"Gupta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cathryn R.","family":"Cadwell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ghiabe","family":"Guibinga","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shirley","family":"Phillips","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tony","family":"Del Rio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fatih","family":"Ozsolak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denise","family":"Imai-Leonard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lingling","family":"Kong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana J.","family":"Laird","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akos","family":"Herzeg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charlotte J.","family":"Sumner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5232-809X","authenticated-orcid":false,"given":"Tippi C.","family":"MacKenzie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2024.101263_bib1","doi-asserted-by":"crossref","first-page":"S36","DOI":"10.1002\/jcph.2127","article-title":"Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting)","volume":"62","author":"Herzeg","year":"2022","journal-title":"J.\u00a0Clin. Pharmacol."},{"key":"10.1016\/j.omtm.2024.101263_bib2","article-title":"In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application","volume":"5","author":"Almeida-Porada","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101263_bib3","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1097\/GRF.0000000000000655","article-title":"Prenatal Gene Therapy","volume":"64","author":"Schwab","year":"2021","journal-title":"Clin. Obstet. Gynecol."},{"key":"10.1016\/j.omtm.2024.101263_bib4","doi-asserted-by":"crossref","first-page":"1206","DOI":"10.1002\/jgm.630","article-title":"Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease","volume":"6","author":"Shen","year":"2004","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.omtm.2024.101263_bib5","doi-asserted-by":"crossref","first-page":"3954","DOI":"10.1096\/fj.201801391R","article-title":"Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques","volume":"33","author":"Chan","year":"2019","journal-title":"FASEB J."},{"key":"10.1016\/j.omtm.2024.101263_bib6","doi-asserted-by":"crossref","first-page":"1843","DOI":"10.1016\/j.ymthe.2017.04.003","article-title":"In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model","volume":"25","author":"Mattar","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib7","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1016\/j.addr.2020.11.002","article-title":"Delivery technologies for in utero gene therapy","volume":"169","author":"Palanki","year":"2021","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.omtm.2024.101263_bib8","doi-asserted-by":"crossref","first-page":"2150","DOI":"10.1056\/NEJMoa2200587","article-title":"In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease","volume":"387","author":"Cohen","year":"2022","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib9","doi-asserted-by":"crossref","first-page":"e41","DOI":"10.1097\/AOG.0000000000001952","volume":"129","year":"2017","journal-title":"Obstet. Gynecol."},{"key":"10.1016\/j.omtm.2024.101263_bib10","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abb6871","article-title":"Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA","volume":"13","author":"Kong","year":"2021","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib11","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1002\/acn3.779","article-title":"Neurofilament as a potential biomarker for spinal muscular atrophy","volume":"6","author":"Darras","year":"2019","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"10.1016\/j.omtm.2024.101263_bib12","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2024.101263_bib13","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1038\/gt.2011.216","article-title":"Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems","volume":"20","author":"Mattar","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib14","doi-asserted-by":"crossref","first-page":"323","DOI":"10.2174\/1566523054065039","article-title":"Immune responses to adeno-associated virus vectors","volume":"5","author":"Zaiss","year":"2005","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib15","doi-asserted-by":"crossref","first-page":"7875","DOI":"10.1128\/JVI.00649-08","article-title":"Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle","volume":"82","author":"Penaud-Budloo","year":"2008","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2024.101263_bib16","doi-asserted-by":"crossref","first-page":"3495","DOI":"10.1128\/JVI.77.6.3495-3504.2003","article-title":"Genetic fate of recombinant adeno-associated virus vector genomes in muscle","volume":"77","author":"Schnepp","year":"2003","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2024.101263_bib17","doi-asserted-by":"crossref","first-page":"14793","DOI":"10.1128\/JVI.79.23.14793-14803.2005","article-title":"Characterization of adeno-associated virus genomes isolated from human tissues","volume":"79","author":"Schnepp","year":"2005","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2024.101263_bib18","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1038\/mt.2008.76","article-title":"Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection","volume":"16","author":"Zincarelli","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib19","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.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib20","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: A Nonrandomized Controlled Trial","volume":"77","author":"Mendell","year":"2020","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.omtm.2024.101263_bib21","doi-asserted-by":"crossref","first-page":"1677","DOI":"10.1038\/sj.mt.6300219","article-title":"Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice","volume":"15","author":"Sabatino","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib22","doi-asserted-by":"crossref","first-page":"2714","DOI":"10.1182\/blood-2004-02-0627","article-title":"Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy","volume":"104","author":"Waddington","year":"2004","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101263_bib23","doi-asserted-by":"crossref","first-page":"2123","DOI":"10.1016\/j.ymthe.2019.08.017","article-title":"Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice","volume":"27","author":"Rashnonejad","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib24","doi-asserted-by":"crossref","first-page":"1317","DOI":"10.1038\/s41591-018-0106-7","article-title":"Fetal gene therapy for neurodegenerative disease of infants","volume":"24","author":"Massaro","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib25","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1038\/s41586-020-2835-2","article-title":"Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA","volume":"587","author":"Wolter","year":"2020","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2024.101263_bib26","doi-asserted-by":"crossref","first-page":"4291","DOI":"10.1038\/s41467-021-24443-8","article-title":"In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease","volume":"12","author":"Bose","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2024.101263_bib27","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1186\/s42826-022-00128-1","article-title":"Role of animal models in biomedical research: a review","volume":"38","author":"Mukherjee","year":"2022","journal-title":"Lab. Anim. Res."},{"key":"10.1016\/j.omtm.2024.101263_bib28","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/j.tibtech.2008.02.002","article-title":"Biomedical applications of sheep models: from asthma to vaccines","volume":"26","author":"Scheerlinck","year":"2008","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtm.2024.101263_bib29","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1146\/annurev-animal-030117-014637","article-title":"Lessons from the Barn to the Operating Suite: A Comprehensive Review of Animal Models for Fetal Surgery","volume":"6","author":"Kabagambe","year":"2018","journal-title":"Annu. Rev. Anim. Biosci."},{"key":"10.1016\/j.omtm.2024.101263_bib30","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1038\/s41591-021-01483-7","article-title":"Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue","volume":"27","author":"Thomsen","year":"2021","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib31","doi-asserted-by":"crossref","first-page":"1971","DOI":"10.1038\/mt.2011.157","article-title":"Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders","volume":"19","author":"Bevan","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib32","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib33","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1089\/hum.2020.167","article-title":"Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology","volume":"31","author":"Hordeaux","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib34","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtm.2018.06.004","article-title":"Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques","volume":"10","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101263_bib35","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.omtm.2018.06.003","article-title":"Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques","volume":"10","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101263_bib36","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1038\/s41434-021-00305-2","article-title":"Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets","volume":"29","author":"Rich","year":"2022","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib37","doi-asserted-by":"crossref","first-page":"9585","DOI":"10.1073\/pnas.160274797","article-title":"The human VASA gene is specifically expressed in the germ cell lineage","volume":"97","author":"Castrillon","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2024.101263_bib38","series-title":"Cellular and Molecular Immunology","author":"Abbas","year":"2018"},{"key":"10.1016\/j.omtm.2024.101263_bib39","doi-asserted-by":"crossref","first-page":"2225","DOI":"10.1172\/JCI31659","article-title":"Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy","volume":"117","author":"Deichmann","year":"2007","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2024.101263_bib40","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":"Hacein-Bey-Abina","year":"2008","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2024.101263_bib41","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1056\/NEJM200301163480314","article-title":"A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency","volume":"348","author":"Hacein-Bey-Abina","year":"2003","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib42","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T\u00a0cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.omtm.2024.101263_bib43","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.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2024.101263_bib44","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1038\/nm1393","article-title":"Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1","volume":"12","author":"Ott","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib45","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1056\/NEJMoa2211075","article-title":"Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A","volume":"388","author":"Mahlangu","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib46","doi-asserted-by":"crossref","first-page":"4266","DOI":"10.1182\/bloodadvances.2021006419","article-title":"Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy","volume":"6","author":"Guillou","year":"2022","journal-title":"Blood Adv."},{"key":"10.1016\/j.omtm.2024.101263_bib47","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","article-title":"Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy","volume":"74","author":"Chand","year":"2021","journal-title":"J.\u00a0Hepatol."},{"key":"10.1016\/j.omtm.2024.101263_bib48","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1089\/hum.2020.182","article-title":"Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy","volume":"31","author":"Wilson","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib49","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1038\/s41587-020-0642-9","volume":"38","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2024.101263_bib50","series-title":"Food and Drug Administration (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) Meeting #70; Toxicity Risks of Adeno-Associated Virus (AAV) Vectors for Gene Therapy","year":"2021"},{"key":"10.1016\/j.omtm.2024.101263_bib51","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0171132","article-title":"Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep","volume":"12","author":"Davey","year":"2017","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2024.101263_bib52","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1089\/hum.2010.007","article-title":"Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep","volume":"22","author":"David","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib53","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1530\/REP-06-0042","article-title":"Factors affecting birth weight in sheep: maternal environment","volume":"133","author":"Gardner","year":"2007","journal-title":"Reproduction"},{"key":"10.1016\/j.omtm.2024.101263_bib54","doi-asserted-by":"crossref","first-page":"612","DOI":"10.2527\/2004.822612x","article-title":"Relationship between litter birth weight and litter size in six breeds of sheep","volume":"82","author":"Freetly","year":"2004","journal-title":"J.\u00a0Anim. Sci."},{"key":"10.1016\/j.omtm.2024.101263_bib55","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1007\/s12015-016-9670-8","article-title":"Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in\u00a0Vivo Cell Tracking Experiments","volume":"12","author":"Ansari","year":"2016","journal-title":"Stem Cell Rev. Rep."},{"key":"10.1016\/j.omtm.2024.101263_bib56","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1016\/j.theriogenology.2007.09.021","article-title":"The pregnant sheep as a model for human pregnancy","volume":"69","author":"Barry","year":"2008","journal-title":"Theriogenology"},{"key":"10.1016\/j.omtm.2024.101263_bib57","doi-asserted-by":"crossref","first-page":"2294","DOI":"10.1056\/NEJMc2212912","article-title":"Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy","volume":"388","author":"Bonnemann","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib58","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1177\/01926233221101309","article-title":"Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques","volume":"50","author":"Meseck","year":"2022","journal-title":"Toxicol. Pathol."},{"key":"10.1016\/j.omtm.2024.101263_bib59","doi-asserted-by":"crossref","first-page":"1979","DOI":"10.1016\/j.ymthe.2023.03.032","article-title":"Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy","volume":"31","author":"Logan","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib60","first-page":"7","article-title":"Meiosis and differentiation of mouse germ cells","volume":"38","author":"McLaren","year":"1984","journal-title":"Symp. Soc. Exp. Biol."},{"key":"10.1016\/j.omtm.2024.101263_bib61","first-page":"39","article-title":"[Kinetics of prespermatogenesis and spermatogenesis]","volume":"68","author":"Hilscher","year":"1974","journal-title":"Verhandlungen Anat. Ges."},{"key":"10.1016\/j.omtm.2024.101263_bib62","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":"10.1016\/j.omtm.2024.101263_bib63","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1038\/sj.gt.3301541","article-title":"Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors","volume":"8","author":"Donsante","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib64","series-title":"The FIGO Manual of Human Reproduction. Vol 1. Reproductive Physiology","author":"Mastroianni","year":"1990"},{"key":"10.1016\/j.omtm.2024.101263_bib65","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3001522","article-title":"Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy","volume":"2","author":"Gentner","year":"2010","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib66","doi-asserted-by":"crossref","first-page":"1409","DOI":"10.1002\/pd.6228","article-title":"Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy","volume":"42","author":"Schwab","year":"2022","journal-title":"Prenat. Diagn."},{"key":"10.1016\/j.omtm.2024.101263_bib67","doi-asserted-by":"crossref","first-page":"1381","DOI":"10.1038\/s41591-022-01866-4","article-title":"Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial","volume":"28","author":"Strauss","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib68","first-page":"E538","article-title":"Metabolic and circulatory studies of fetal lamb at midgestation","volume":"250","author":"Bell","year":"1986","journal-title":"Am. J. Physiol."},{"key":"10.1016\/j.omtm.2024.101263_bib69","doi-asserted-by":"crossref","first-page":"379","DOI":"10.3181\/00379727-195-43158AA","article-title":"Uterine blood flow, oxygen and glucose uptakes at mid-gestation in the sheep","volume":"195","author":"Molina","year":"1990","journal-title":"Proc. Soc. Exp. Biol. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib70","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/nchem.2301","article-title":"Optimization of a whole-cell biocatalyst by employing genetically encoded product sensors inside nanolitre reactors","volume":"7","author":"Meyer","year":"2015","journal-title":"Nat. Chem."},{"key":"10.1016\/j.omtm.2024.101263_bib71","doi-asserted-by":"crossref","first-page":"1379","DOI":"10.1038\/nprot.2010.87","article-title":"Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing","volume":"5","author":"Paruzynski","year":"2010","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omtm.2024.101263_bib72","doi-asserted-by":"crossref","first-page":"1431","DOI":"10.1038\/nm.2057","article-title":"Comprehensive genomic access to vector integration in clinical gene therapy","volume":"15","author":"Gabriel","year":"2009","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101263_bib73","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1089\/104303401750148649","article-title":"Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples","volume":"12","author":"Schmidt","year":"2001","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib74","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1016\/j.omtn.2016.12.001","article-title":"GENE-IS: Time-Efficient and Accurate Analysis of Viral Integration Events in Large-Scale Gene Therapy Data","volume":"6","author":"Afzal","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2024.101263_bib75","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/j.csbj.2015.10.006","article-title":"A Graph Based Framework to Model Virus Integration Sites","volume":"14","author":"Fronza","year":"2016","journal-title":"Comput. Struct. Biotechnol. J."},{"key":"10.1016\/j.omtm.2024.101263_bib76","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0024247","article-title":"Analyzing the number of common integration sites of viral vectors--new methods and computer programs","volume":"6","author":"Abel","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2024.101263_bib77","doi-asserted-by":"crossref","first-page":"1584","DOI":"10.1128\/JVI.77.2.1584-1588.2003","article-title":"Common sites of retroviral integration in mouse hematopoietic tumors identified by high-throughput, single nucleotide polymorphism-based mapping and bacterial artificial chromosome hybridization","volume":"77","author":"Shen","year":"2003","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2024.101263_bib78","doi-asserted-by":"crossref","DOI":"10.1093\/bib\/bbab220","article-title":"COSMIC Cancer Gene Census 3D database: understanding the impacts of mutations on cancer targets","volume":"22","author":"Alsulami","year":"2021","journal-title":"Briefings Bioinf."},{"key":"10.1016\/j.omtm.2024.101263_bib79","doi-asserted-by":"crossref","first-page":"1457","DOI":"10.1073\/pnas.0507496103","article-title":"Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T\u00a0cells","volume":"103","author":"Recchia","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2024.101263_bib80","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1038\/sj.mt.6300394","article-title":"Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cells","volume":"16","author":"Hargrove","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101263_bib81","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.36245","article-title":"The human leukemia virus HTLV-1 alters the structure and transcription of host chromatin in cis","volume":"7","author":"Melamed","year":"2018","journal-title":"Elife"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124000792?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124000792?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,9,15]],"date-time":"2024-09-15T11:05:29Z","timestamp":1726398329000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050124000792"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,6]]},"references-count":81,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2024,6]]}},"alternative-id":["S2329050124000792"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101263","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2024,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101263","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Author(s).","name":"copyright","label":"Copyright"}],"article-number":"101263"}}