Biblio

Author Title [ Type(Desc)] Year
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Journal Article
Bradford KL, Liu S, Krajinovic M, Ansari M, Garabedian E, Tse J, Wang X, Shaw KL, H Gaspar B, Candotti F, et al. Busulfan Pharmacokinetics in ADA SCID Gene Therapy. Biol Blood Marrow Transplant. 2020.
Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, H Gaspar B, Notarangelo LD, Grunebaum E. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2018.
Al-Herz W, Chu JI, van der Spek J, Raghupathy R, Massaad MJ, Keles S, Biggs CM, Cockerton L, Chou J, Dbaibo G, et al. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016.
Wehr C, Gennery AR, Lindemans C, Schulz A, Hoenig M, Marks R, Recher M, Gruhn B, Holbro A, Heijnen I, et al. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol. 2015.
Abina SHacein-Bey, H Gaspar B, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, et al. Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome. JAMA. 2015;313(15):1550-1563.
Booth C, H Gaspar B, Thrasher AJ. Treating Immunodeficiency through HSC Gene Therapy. Trends Mol Med. 2016.