Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.

E, Mamcarz; S, Zhou; T, Lockey; H, Abdelsamed; SJ, Cross; G, Kang; Z, Ma; J, Condori; J, Dowdy; B, Triplett; C, Li; G, Maron; Becerra JC, Aldave; JA, Church; E, Dokmeci; JT, Love; Matta Ain AC, da; der Watt H, van; X, Tang; W, Janssen; BY, Ryu; Ravin SS, De; MJ, Weiss; B, Youngblood; JR, Long-Boyle; S, Gottschalk; MM, Meagher; HL, Malech; JM, Puck; MJ, Cowan; BP, Sorrentino

doi:10.1056/nejmoa1815408

Public

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.

Shared by NobleBlocks on Apr 1, 2019 • 12:00 AM UTC

Authors:

Mamcarz E

Zhou S

Lockey T

Abstract

<h4>Background</h4>Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy...

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