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Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates

Hematopoietic stem cell gene therapy has been successfully used for a number of genetic diseases and is also being explored for HIV. However, toxicity of the conditioning regimens has been a major concern. Here we compared current conditioning approaches in a clinically relevant nonhuman primate mod...

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Detalles Bibliográficos
Autores principales: Murray, Jason, Einhaus, Teresa, Venkataraman, Rasika, Radtke, Stefan, Zhen, Anjie, Carrillo, Mayra A., Kitchen, Scott G., Peterson, Christopher W., Kiem, Hans-Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10415663/
https://www.ncbi.nlm.nih.gov/pubmed/37575091
http://dx.doi.org/10.1016/j.omtm.2023.07.006

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