Retroviral vector for gene therapy of XXXX .

@entity1 @entity10191 @entity19 @entity1112 @entity42327 @entity915 @entity58 @entity74 @entity22416
@entity915 ( @entity915 ) is a @entity74 characterized by profound impairment of cell-mediated and humoral immunity. Affected @entity1 die of recurrent infections within 2 years of birth unless rescued by allogeneic transplantation from a suitable donor. Recently, the @entity74 responsible for @entity1112 @entity915 has been identified as a mutation in the gamma chain of the IL-2 receptor, a protein also shared by the @entity58 and @entity10191 receptors and therefore now denoted the @entity42327 ( @entity42327 ). We report here the development of a high-titer amphotropic retroviral vector for transfer of @entity42327 . This vector was used to transfer a copy of the @entity42327 cDNA to @entity19 3T3 fibroblasts, @entity22416 -enriched hematopoietic progenitor cells obtained from bone marrow and umbilical cord blood of normal donors, and to transplanted @entity19 bone marrow progenitors. @entity19 3T3 cells transduced by the retroviral vector were analyzed by Southern blot hybridization and Western transfer. Southern analysis confirmed the integration of unrearranged proviral DNA, and Western blot analysis demonstrated the expression of @entity42327 protein. @entity22416 -enriched cells were infected with viral vectors bearing @entity42327 and grown in methylcellulose media. Individual colonies and pools of cells were analyzed 2 weeks later by polymerase chain reaction assay, which confirmed the proviral marking. The vector was also used to transfer a copy of the @entity42327 cDNA to @entity19 bone marrow cells in a transplantation model. Infected marrow was transplanted into syngeneic Balb/c @entity19 .(ABSTRACT TRUNCATED AT 250 WORDS)
