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Adenosine deaminase (ADA) deficiency is a good gene therapy model for targeting a rare population of pluripotent hematopoietic stem cells capable of self-renewal. We present evidence for the highly efficient gene transfer and sustained expression of hum an ADA in human primitive hematopoietic progenitors using retroviral supernatant with a supportive stromal layer. A stem cell-enriched (CD34+ ) fraction was also successfully transduced. Duchenne muscular dystrophy (DMD) is also a good model for somatic gene therapy. Two of the challenges presented by this model are the large size of the gene and the large number of target cells. Germline gene transfer and correction of the phenotype has been demonstrated in transgenic mdx mice using both a full-length and a truncated form of the dystrophin cDNA. We present here a deletion mutagenesis strategy to truncate the dystrophin cDNA such that it can be accommodated by retroviral and adenoviral vectors useful for somatic gene therapy.<\/jats:p>","DOI":"10.1098\/rstb.1993.0019","type":"journal-article","created":{"date-parts":[[2006,12,18]],"date-time":"2006-12-18T19:03:09Z","timestamp":1166468589000},"page":"217-224","source":"Crossref","is-referenced-by-count":5,"title":["Gene transfer therapy for heritable disease: cell and expression targeting"],"prefix":"10.1098","volume":"339","author":[{"given":"Kohnoske","family":"Mitani","sequence":"first","affiliation":[{"name":"Howard Hughes Medical Institute, Baylor College of Medicine, One Baylor Plaza, Houston, Texas 77030, U. S. A."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paula R.","family":"Clemens","sequence":"additional","affiliation":[{"name":"Institute for Molecular Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, Texas 77030, U. S. 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