Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells

Targeted chromosomal insertion of large genetic payloads in human cells leverages and broadens synthetic biology and genetic therapy efforts. Yet, obtaining large-scale gene knock-ins remains particularly challenging especially in hard-to-transfect stem and progenitor cells. Here, fully viral gene-d...

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Detalles Bibliográficos
Autores principales: Tasca, Francesca, Brescia, Marcella, Wang, Qian, Liu, Jin, Janssen, Josephine M, Szuhai, Karoly, Gonçalves, Manuel A F V
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Synthetic Biology and Bioengineering
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9303392/
https://www.ncbi.nlm.nih.gov/pubmed/35776127
http://dx.doi.org/10.1093/nar/gkac567

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9303392/
https://www.ncbi.nlm.nih.gov/pubmed/35776127
http://dx.doi.org/10.1093/nar/gkac567

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