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

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 giv...

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Publicat a:N Engl J Med
Autors principals: Mamcarz, E., Zhou, S., Lockey, T., Abdelsamed, H., Cross, S.J., Kang, G., Ma, Z., Condori, J., Dowdy, J., Triplett, B., Li, C., Maron, G., Aldave Becerra, J.C., Church, J.A., Dokmeci, E., Love, J.T., Ain, A.C. da Matta, van der Watt, H., Tang, X., Janssen, W., Ryu, B.Y., De Ravin, S.S., Weiss, M.J., Youngblood, B., Long‑Boyle, J.R., Gottschalk, S., Meagher, M.M., Malech, H.L., Puck, J.M., Cowan, M.J., Sorrentino, B.P.
Format: Artigo
Idioma:Inglês
Publicat: 2019
Matèries:
Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6636624/
https://ncbi.nlm.nih.gov/pubmed/30995372
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1815408
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