Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Adrenoleukodystrophy is an X-linked genetic disease that is caused by a defect in the gene ABCD1 (ATP-binding cassette, subfamily D, member 1), which encodes the peroxisomal ABC half-transporter ALD protein. Mutations in ABCD1 result in abnormal breakdown of very-long-chain fatty acids, a process…

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