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

• Main Authors: Jean-Paul (Author), Dominique L Ouellet (Author), Xavier Barbeau (Author), Joël Rousseau (Author), Pierre Chapdelaine (Author), Patrick Lagüe (Author), Jacques P Tremblay (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...