Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

摘要

H:/aar/ntenvovciNRPobI/DCCAAR9940491_ dox-31/1012014 The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy. 54a