Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408.
N Engl J Med. 2019.
PMID: 30995372
Free PMC article.
Clinical Trial.