High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells

Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle wasting disorder arising from mutations in the ~2.4 Mb dystrophin-encoding DMD gene. RNA-guided CRISPR-Cas9 nucleases (RGNs) are opening new DMD therapeutic routes whose bottlenecks include delivering sizable RGN complexes for assessing th...

Descripción completa

Detalles Bibliográficos
Autores principales: Brescia, Marcella, Janssen, Josephine M., Liu, Jin, Gonçalves, Manuel A. F. V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7226760/
https://www.ncbi.nlm.nih.gov/pubmed/32252479
http://dx.doi.org/10.3390/cells9040869

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7226760/
https://www.ncbi.nlm.nih.gov/pubmed/32252479
http://dx.doi.org/10.3390/cells9040869

Ejemplares similares