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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Detalles Bibliográficos
Autores principales: Li, Chang, Psatha, Nikoletta, Gil, Sucheol, Wang, Hongjie, Papayannopoulou, Thalia, Lieber, André
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6054697/
https://www.ncbi.nlm.nih.gov/pubmed/30038942
http://dx.doi.org/10.1016/j.omtm.2018.04.008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6054697/
https://www.ncbi.nlm.nih.gov/pubmed/30038942
http://dx.doi.org/10.1016/j.omtm.2018.04.008

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