Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.
N Engl J Med. 2022.
PMID: 34891223
Clinical Trial.