Biblio
CRISPR-Cas9 Editing of the and Promoters to Treat Sickle Cell Disease. N Engl J Med. 2023;389(9):820-832.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Outcomes in Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia. Transplant Cell Ther. 2021.