CRISPR/Cas9-mediated genome editing tagged posts

Correction of RPGR mutation in patient iPSC line.

CUMC and Uni of Iowa scientists have used CRISPR, to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide. It marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue.

“Our vision is to develop a personalized approach to treating eye disease,” says A/Prof Stephen Tsang, MD, PhD. “We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible...

Members of the UT Southwestern team whose research successfully halted progression of a form of muscular dystrophy in mice included (l-r) Dr. Chengzu Long, Dr. Eric Olson, Dr. Rhonda Bassel-Duby, Dr. Leonela Amoasii, John Shelton, and Alex Mireault. Credit: UT Southwestern Medical Center

Using a new gene-editing technique, UT Southwestern Medical Center scientists stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up in DMD patients, this technique could lead to one of the first successful genome editing Rx’s.

DMD, the most common and severe form of muscular dystrophy among boys, is characterized by progressive muscle degeneration and weakness. It is caused by mutations in the X-linked DMD gene that encodes the protein dystrophin...