Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII

Hematopoietic stem and progenitor cell (HSPC) lentiviral gene therapy is a promising strategy toward a lifelong cure for hemophilia A (HA). The primary risks associated with this approach center on the requirement for pre-transplantation conditioning necessary to make space for, and provide immune s...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Russell, Athena L., Prince, Chengyu, Lundgren, Taran S., Knight, Kristopher A., Denning, Gabriela, Alexander, Jordan S., Zoine, Jaquelyn T., Spencer, H. Trent, Chandrakasan, Shanmuganathan, Doering, Christopher B.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8181577/
https://ncbi.nlm.nih.gov/pubmed/34141826
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.04.016
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