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Current clinical results indicate that (a) both umbilical cord blood and neonatal bone marrow HSC can be transduced with murine retroviral-based vectors, (b) the transduced HSC can engraft in nonmyeloablated patients, (c) the frequency of HSC transduction\/engraftment is low (1\/10,000), (d) an in vivo selective advantage can exist for transduced T lymphoid progeny, and (e) the transduced ADA gene is not expressed in nondividing T lymphocytes. Improving the clinical results of gene therapy for ADA deficiency and other genetic diseases involving HSC will require (a) developing new vectors that express the transduced gene in nondividing cells and (b) increasing the frequency of stable HSC transduction. <\/jats:p>","DOI":"10.1146\/annurev.med.51.1.33","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T11:45:04Z","timestamp":1027770304000},"page":"33-47","source":"Crossref","is-referenced-by-count":27,"title":["Gene Therapy for Adenosine Deaminase Deficiency"],"prefix":"10.1146","volume":"51","author":[{"given":"Robertson","family":"Parkman","sequence":"first","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenneth","family":"Weinberg","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gay","family":"Crooks","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jan","family":"Nolta","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neena","family":"Kapoor","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Donald","family":"Kohn","sequence":"additional","affiliation":[{"name":"Division of Research Immunology\/Bone Marrow Transplantation, Childrens Hospital Los Angeles and the Department of Pediatrics, University of Southern California, School of Medicine, Los Angeles, California, 90027;"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"22","reference":[{"key":"b1","doi-asserted-by":"publisher","DOI":"10.1126\/science.6093246"},{"key":"b2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.26.15388"},{"key":"b3","doi-asserted-by":"crossref","unstructured":"Isner JM, Baumgartner I, Rauh G, et al. 1998. 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