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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Blood Adv
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Hematology 2021
Pynciau:
Gene Therapy
Mynediad Ar-lein: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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