Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

We have developed an in vivo hemopoietic stem cell (HSC) gene therapy approach without the need for myelosuppressive conditioning and autologous HSC transplantation. It involves HSC mobilization and IV injection of a helper-dependent adenovirus HDAd5/35(++) vector system. The current mobilization re...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Li, Chang, Goncalves, Kevin A., Raskó, Tamás, Pande, Amit, Gil, Sucheol, Liu, Zhinan, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Davis, John C., Kiem, Hans-Peter, Lieber, André
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2021
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7948287/
https://ncbi.nlm.nih.gov/pubmed/33646305
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020003714
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