Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
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N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.
N Engl J Med. 2017.
PMID: 28976817
Free PMC article.
Clinical Trial.