Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases

With the development of CRISPR-Cas9-mediated gene-editing technologies, correction of disease-causing mutations has become possible. However, current gene-correction strategies preclude mutation repair in post-mitotic cells of human tissues, and a unique repair strategy must be designed and tested f...

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Detalles Bibliográficos
Autores principales: Hu, Jingjie, Bourne, Rebecca A., McGrath, Barbara C., Lin, Alice, Pei, Zifei, Cavener, Douglas R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571108/
https://www.ncbi.nlm.nih.gov/pubmed/33892188
http://dx.doi.org/10.1016/j.ymthe.2021.04.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571108/
https://www.ncbi.nlm.nih.gov/pubmed/33892188
http://dx.doi.org/10.1016/j.ymthe.2021.04.017

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