Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

Hematopoietic stem-cell gene therapy is a promising treatment of X-linked severe combined immunodeficiency disease (SCID-X1), but currently, it requires recipient conditioning, extensive cell manipulation, and sophisticated facilities. With these limitations in mind, we explored a simpler therapeuti...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Humbert, Olivier, Chan, Frieda, Rajawat, Yogendra S., Torgerson, Troy R., Burtner, Christopher R., Hubbard, Nicholas W., Humphrys, Daniel, Norgaard, Zachary K., O’Donnell, Patricia, Adair, Jennifer E., Trobridge, Grant D., Scharenberg, Andrew M., Felsburg, Peter J., Rawlings, David J., Kiem, Hans-Peter
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2018
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5942001/
https://ncbi.nlm.nih.gov/pubmed/29720491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2018016451
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