Gene therapy vectors for adrenal cerebral white matter dystrophy and adrenal spinal neuropathy

摘要

The present invention provides compositions ccomprising 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.