NEW YORK (GenomeWeb) – Scientists at the University of California, Los Angeles have received a two-year $2.2 million grant from the California Institute of Regenerative Medicine to evaluate the feasibility of using CRISPR/Cas9 in gene therapies for Duchenne Muscular Dystrophy.
Led by April Pyle and Melissa Spencer of UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and UCLA nanomaterials expert Huan Meng, the researchers will use two different CRISPR/Cas9-based approaches to deliver the gene-editing platform in a mouse model of DMD and use it to correct the mutations responsible for the disease.
Mutations in the dystrophin gene can result in low production of the protein, leading to muscle degeneration. While several gene therapies are in clinical trials, there are no current therapies that address the root cause of the genetic disease. The researchers will pursue two paths to correcting mutations in the dystrophin gene: by delivering CRISPR/Cas9 to muscle stem cells and by editing stem cells ex vivo and distributing them around the body.
Since the beginning of the year, several labs, including Pyle's, have announced success in using CRISPR/Cas9 to edit some of the mutations that cause DMD. Pyle and Spencer published a study in Cell Stem Cell in April showing gene editing of dystrophin mutations in patient-derived induced pluripotent stem cells.
"Once we developed the CRISPR/Cas9 platform for Duchenne mutations, we knew our next challenge would be to correct the muscle stem cells that reside throughout approximately 40 percent of the body that is made of muscle tissues,” Pyle said in a statement.
The results of the studies could indicate whether these are viable methods for gene therapies for DMD, the researchers added.