{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2023,11,15]],"date-time":"2023-11-15T09:14:12Z","timestamp":1700039652805},"reference-count":39,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2003,8,18]],"date-time":"2003-08-18T00:00:00Z","timestamp":1061164800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Journal of Medical Virology"],"published-print":{"date-parts":[[2003,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Lentiviral vectors are prime candidate vectors for gene transfer into dividing and non\u2010dividing cells, including neuronal cells and stem cells. For safety, HIV\u20102 lentiviral vectors may be better suited for gene transfer in humans than HIV\u20101 lentiviral vectors. HIV\u20102 vectors cross\u2010packaged in HIV\u20101 cores may be even safer. Demonstration of the efficacy of these vectors in disease models will validate their usefulness. Parkinson's disease and Fabry disease provide excellent models for validation. Parkinson's disease is a focal degeneration of dopaminergic neurons in the brain with progressive loss of ability to produce the neurotransmitter dopamine. Current treatment entails administration of increasing doses of L\u2010dopa, with attendant toxicity. We explore here the hypothesis that gene transfer of aromatic acid decarboxylase (AADC), a key enzyme in the pathway, will make neuronal cells more efficiently convert L\u2010dopa into dopamine. Fabry disease on the other hand is a monogenic inherited disease, characterized by \u03b1\u2010galactosidase A (AGA) deficiency, resulting in glycolipid accumulation in several cell types, including fibroblasts. Animal models for preclinical investigations of both of these diseases are available. We have designed monocistronic HIV\u20101 and HIV\u20102 vectors with the AADC transgene and monocistronic and bicistronic HIV\u20102 vectors with the AGA and puromycin resistance transgenes. They were packaged with either HIV\u20102 cores or HIV\u20101 cores (hybrid vectors). Gene transfer of AADC gene in neuronal cells imparted the ability on the transduced cells to efficiently convert L\u2010dopa into dopamine. Similarly, the AGA vectors induced Fabry fibroblasts to produce high levels of AGA enzyme and caused rapid clearance of the glycolipids from the cells. Both monocistronic and bicistronic vectors were effective. Thus, the insertion of a second gene downstream in the bicistronic vector was not deleterious. In addition, both the self\u2010packaged vectors and the cross\u2010packaged hybrid vectors were effective in gene transfer. J. Med. Virol. 71:173\u2013182, 2003. \u00a9 2003 Wiley\u2010Liss, Inc.<\/jats:p>","DOI":"10.1002\/jmv.10467","type":"journal-article","created":{"date-parts":[[2003,8,22]],"date-time":"2003-08-22T14:29:20Z","timestamp":1061562560000},"page":"173-182","source":"Crossref","is-referenced-by-count":14,"title":["HIV\u20102 derived lentiviral vectors: Gene transfer in Parkinson's and Fabry disease models in vitro"],"prefix":"10.1002","volume":"71","author":[{"given":"Jenice","family":"D'Costa","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Judith","family":"Harvey\u2010White","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pankaj","family":"Qasba","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Advait","family":"Limaye","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christine R.","family":"Kaneski","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alberta","family":"Davis\u2010Warren","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roscoe O.","family":"Brady","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Krys S.","family":"Bankiewicz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eugene O.","family":"Major","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Suresh K.","family":"Arya","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2003,8,18]]},"reference":[{"key":"e_1_2_1_2_1","doi-asserted-by":"crossref","first-page":"2581","DOI":"10.1128\/jvi.70.4.2581-2585.1996","article-title":"High efficiency gene transfer into CD34+ cells with a human immunodeficiency viral type 1\u2010based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G","volume":"70","author":"Akkina RK","year":"1996","journal-title":"J Virol"},{"key":"e_1_2_1_3_1","doi-asserted-by":"publisher","DOI":"10.1089\/aid.1998.14.59"},{"key":"e_1_2_1_4_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.285.5428.674"},{"key":"e_1_2_1_5_1","doi-asserted-by":"publisher","DOI":"10.1089\/aid.1993.9.839"},{"key":"e_1_2_1_6_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.9.4486"},{"key":"e_1_2_1_7_1","first-page":"1205","article-title":"Fusogenicity of mutant and chimeric proviruses derived from molecular clones of cytopathic and non\u2010cytopathic human immunodeficiency virus type 2","volume":"6","author":"Arya SK","year":"1993","journal-title":"J Acquir Immun Def Synd"},{"key":"e_1_2_1_8_1","first-page":"515S","article-title":"HIV\u20102 inhibits HIV\u20101","volume":"10","author":"Arya SK","year":"1994","journal-title":"AIDS Res Hum Retrovir"},{"key":"e_1_2_1_9_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.1998.9.9-1371"},{"key":"e_1_2_1_10_1","doi-asserted-by":"publisher","DOI":"10.1385\/1-59259-393-3:219"},{"key":"e_1_2_1_11_1","doi-asserted-by":"publisher","DOI":"10.1006\/exnr.2000.7408"},{"key":"e_1_2_1_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM196705252762101"},{"key":"e_1_2_1_13_1","doi-asserted-by":"crossref","first-page":"5191","DOI":"10.1128\/JVI.73.6.5191-5195.1999","article-title":"Potent inhibition of human immunodeficiency virus type 1 (HIV\u20101) gene expression and virus production by an HIV\u20102 Tat activation\u2010response RNA decoy","volume":"73","author":"Browning CM","year":"1999","journal-title":"J Virol"},{"key":"e_1_2_1_14_1","doi-asserted-by":"publisher","DOI":"10.1099\/0022-1317-82-2-425"},{"key":"e_1_2_1_15_1","first-page":"2741","volume-title":"The metabolic and molecular basis of inherited diseases","author":"Desnick RJ","year":"1995"},{"key":"e_1_2_1_16_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.1995.6.2-177"},{"key":"e_1_2_1_17_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.051634498"},{"key":"e_1_2_1_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/83324"},{"key":"e_1_2_1_19_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature00900"},{"key":"e_1_2_1_20_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.97.12.6797"},{"key":"e_1_2_1_21_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.290.5492.767"},{"key":"e_1_2_1_22_1","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1128\/jvi.64.2.890-901.1990","article-title":"Molecular characterization of an attenuated human immunodeficiency virus type 2 isolate","volume":"64","author":"Kumar P","year":"1990","journal-title":"J Virol"},{"key":"e_1_2_1_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0306-4522(98)00741-6"},{"key":"e_1_2_1_24_1","first-page":"488","article-title":"Lentiviral vectors: progress and potential","volume":"2","author":"Lever AM","year":"2000","journal-title":"Curr Opin Mol Ther"},{"key":"e_1_2_1_25_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.7915856"},{"key":"e_1_2_1_26_1","doi-asserted-by":"crossref","first-page":"4544","DOI":"10.1128\/jvi.71.6.4544-4554.1997","article-title":"Efficient encapsidation of human immunodeficiency viral vectors and further characterization of cis elements required for encapsidation","volume":"71","author":"McBride MS","year":"1997","journal-title":"J Virol"},{"key":"e_1_2_1_27_1","doi-asserted-by":"publisher","DOI":"10.1128\/jvi.71.5.4133-4137.1997"},{"key":"e_1_2_1_28_1","first-page":"437","volume-title":"Retroviruses","author":"Miller AD","year":"1997"},{"key":"e_1_2_1_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/0005-2760(92)90129-J"},{"key":"e_1_2_1_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0065-3527(00)55020-9"},{"key":"e_1_2_1_31_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.272.5259.263"},{"key":"e_1_2_1_32_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.6.2540"},{"key":"e_1_2_1_33_1","doi-asserted-by":"crossref","first-page":"6527","DOI":"10.1128\/JVI.72.8.6527-6536.1998","article-title":"Identification of a human immunodeficiency virus type 2 (HIV\u20102) encapsidation determinant and transduction of nondividing human cells by HIV\u20102 based lentivirus vectors","volume":"72","author":"Poeschla E","year":"1998","journal-title":"J Virol"},{"key":"e_1_2_1_34_1","doi-asserted-by":"publisher","DOI":"10.1086\/315010"},{"key":"e_1_2_1_35_1","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1096-9071(199802)54:2<118::AID-JMV9>3.0.CO;2-9"},{"key":"e_1_2_1_36_1","doi-asserted-by":"publisher","DOI":"10.1006\/mthe.2001.0466"},{"key":"e_1_2_1_37_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1012455421014"},{"key":"e_1_2_1_38_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.120177997"},{"key":"e_1_2_1_39_1","doi-asserted-by":"publisher","DOI":"10.1089\/10430349950017671"},{"key":"e_1_2_1_40_1","doi-asserted-by":"publisher","DOI":"10.1038\/nbt0997-871"}],"container-title":["Journal of Medical Virology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fjmv.10467","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jmv.10467","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,8]],"date-time":"2023-10-08T09:40:27Z","timestamp":1696758027000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jmv.10467"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2003,8,18]]},"references-count":39,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2003,10]]}},"alternative-id":["10.1002\/jmv.10467"],"URL":"https:\/\/doi.org\/10.1002\/jmv.10467","archive":["Portico"],"relation":{},"ISSN":["0146-6615","1096-9071"],"issn-type":[{"value":"0146-6615","type":"print"},{"value":"1096-9071","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,8,18]]}}}