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HDAd5/35(++) Adenovirus Vector Expressing Anti-CRISPR Peptides Decreases CRISPR/Cas9 Toxicity in Human Hematopoietic Stem Cells

We generated helper-dependent HDAd5/35(++) adenovirus vectors expressing CRISPR/Cas9 for potential hematopoietic stem cells (HSCs) gene therapy of β-thalassemia and sickle cell disease through re-activation of fetal γ-globin expression (HDAd-globin-CRISPR). The process of CRISPR/Cas9 gene transfer u...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Li, Chang, Psatha, Nikoletta, Gil, Sucheol, Wang, Hongjie, Papayannopoulou, Thalia, Lieber, André
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6054697/
https://ncbi.nlm.nih.gov/pubmed/30038942
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.04.008
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