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.
Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries. Orphanet J Rare Dis. 2024;19(1):46.
Outcomes in Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia. Transplant Cell Ther. 2021.