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In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model
We have recently reported that, after in vivo hematopoietic stem cell/progenitor (HSPC) transduction with HDAd5/35++ vectors, SB100x transposase-mediated γ-globin gene addition achieved 10%–15% γ-globin of adult mouse globin, resulting in significant but incomplete phenotypic correction in a thalass...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | Mol Ther |
|---|---|
| Prif Awduron: | , , , , , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
American Society of Gene & Cell Therapy
2021
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| Pynciau: | |
| Mynediad Ar-lein: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7854285/ https://ncbi.nlm.nih.gov/pubmed/32949495 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.09.001 |
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