Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.
N Engl J Med. 2021.
PMID: 33974366
Free PMC article.
Clinical Trial.