AAV-ABCD1 CONSTRUCTS AND USE FOR TREATMENT OR PREVENTION OF ADRENOLEUKODYSTROPHY (ALD) AND/OR ADRENOMYELONEUROPATHY (AMN)

摘要

The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.