Biblio
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Hematopoietic stem cell transplantation for mucopolysaccharidoses; past, present, and future. Biol Blood Marrow Transplant. 2019.
International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome. J Allergy Clin Immunol. 2021.
Outcome after a cord blood transplantation using busulfan pharmacokinetic targeted myeloablative conditioning for Hurler syndrome. Biol Blood Marrow Transplant. 2020.
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