Related Patent Info
PREVENTION OF MUSCULAR DYSTROPHY BY CRISPR/CAS9-MEDIATED GENE EDITING
| Inventor | OLSON, Eric, N., etc. |
|---|---|
| Assignee | THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM |
| Title of Invention | PREVENTION OF MUSCULAR DYSTROPHY BY CRISPR/CAS9-MEDIATED GENE EDITING |
| Abstract | Provided is a method for correcting a dystrophin gene defect in a patient with Duchenne muscular dystrophy (DMD) through skipping of a mutant exon of the gene by means of CRISPR/Cas9-mediated gene editing, the method being applicable to a muscle cell, a satellite cell or an iPS cell from the DMDpatient. |