Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors

Our goal is the development of in vivo hematopoietic stem cell (HSC) transduction technology with targeted integration. To achieve this, we modified helper-dependent HDAd5/35++ vectors to express a CRISPR/Cas9 specific to the “safe harbor” adeno-associated virus integration site 1 (AAVS1) locus and...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Li, Chang, Mishra, Arpit Suresh, Gil, Sucheol, Wang, Meng, Georgakopoulou, Aphrodite, Papayannopoulou, Thalia, Hawkins, R. David, Lieber, André
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6904827/
https://ncbi.nlm.nih.gov/pubmed/31494053
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.08.006
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