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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Dades bibliogràfiques
Publicat a:Blood Adv
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2018
Matèries:
Gene Therapy
Accés en línia: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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