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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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| Опубликовано в: : | Blood Adv |
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| Главные авторы: | , , , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society of Hematology
2021
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| Предметы: | |
| Online-ссылка: | 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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