Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells

Mutations disrupting the reading frame of the ~2.4 Mb dystrophin-encoding DMD gene cause a fatal X-linked muscle-wasting disorder called Duchenne muscular dystrophy (DMD). Genome editing based on paired RNA-guided nucleases (RGNs) from CRISPR/Cas9 systems has been proposed for permanently repairing...

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Detalles Bibliográficos
Autores principales: Maggio, Ignazio, Liu, Jin, Janssen, Josephine M., Chen, Xiaoyu, Gonçalves, Manuel A. F. V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5109245/
https://www.ncbi.nlm.nih.gov/pubmed/27845387
http://dx.doi.org/10.1038/srep37051

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5109245/
https://www.ncbi.nlm.nih.gov/pubmed/27845387
http://dx.doi.org/10.1038/srep37051

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