Biblio
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency. Sci Transl Med. 2024;16(733):eadh8162.
Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease. J Allergy Clin Immunol Pract. 2023.
Progressive B Cell Loss in Revertant X-SCID. J Clin Immunol. 2020.
Gene Editing in Chronic Granulomatous Disease. Methods Mol Biol. 2019;1982:623-665.
. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019;380(16):1525-1534.
Enhancers Are Major Targets for MLV Vector Integration. J Virol. 2014.