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

Mamcarz, Ewelina; Zhou, Sheng; Lockey, T.; A. Abdelsamed, Hossam; J. Cross, Shane; Kang, G.; Ma, Zhijun; Condori, J.; Dowdy, Jola; Triplett, B.; Li, Chen; M. Maron, Gabriela; C. Aldave Becerra, J.; Church, J.; Dokmeci, E.; Love, J.; C. da Matta Ain, A.; van der Watt, Hedi; Tang, Xing; Janssen, W.; Ryu, B.; de Ravin, S.; Weiss, M.; Youngblood, Ben; Long-Boyle, J.; Gottschalk, S.; Meagher, M.; Malech, H.; Puck, J.; Cowan, M.; Sorrentino, B.

doi:10.1056/NEJMoa1815408

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

Shared by NobleBlocks on Apr 17, 2019 • 12:00 AM UTC

Authors:

Ewelina Mamcarz

Sheng Zhou

T. Lockey

Abstract

BACKGROUND 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 is give...

Subject

Medicine

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