Biblio
Busulfan Pharmacokinetics in ADA SCID Gene Therapy. Biol Blood Marrow Transplant. 2020.
Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2018.
. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016.
Treating Immunodeficiency through HSC Gene Therapy. Trends Mol Med. 2016.
. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol. 2015.
Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome. JAMA. 2015;313(15):1550-1563.