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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...

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發表在:Mol Ther
Main Authors: Li, Chang, Wang, Hongjie, Georgakopoulou, Aphrodite, Gil, Sucheol, Yannaki, Evangelia, Lieber, André
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2021
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在線閱讀: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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