Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

The CRISPR/Cas9 system is a great revolution in biology. This technology allows the modification of genes in vitro and in vivo in a wide variety of living organisms. In most Duchenne muscular dystrophy (DMD) patients, expression of dystrophin (DYS) protein is disrupted because exon deletions result...

Full description

Bibliographic Details
Main Authors: Jean-Paul Iyombe-Engembe, Dominique L Ouellet, Xavier Barbeau, Joël Rousseau, Pierre Chapdelaine, Patrick Lagüe, Jacques P Tremblay
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
Cas9
CRISPR
DMD gene
Duchenne muscular dystrophy
dystrophin
gene therapy
gRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117300148

Similar Items

Similar Items