NEW YORK (GenomeWeb) – Parent Project Muscular Dystrophy today announced it has granted $250,000 to University of Texas Southwestern Medical Center researcher Eric Olson for his study of a CRISPR/Cas9-based treatment for Duchenne muscular dystrophy (DMD).
Olson said in a statement that he would use the funding to test the effectiveness and safety of CRISPR in animal models of DMD. The lab will study dystrophin proteins produced using genome editing in mice and compare them to dystrophin produced by other therapeutic approaches, such as gene transfer.
PPMD is a non-profit organization that focused on finding treatments for DMD, a genetic disorder characterized by a lack of dystrophin protein, which is an essential component of muscle and other tissues. The disease affects approximately one in 5,000 males born in the US, PPMD said.
DMD gene therapy has become an early hotspot in CRISPR/Cas9-based gene therapy. In 2014, Olson showed that his lab could prevent DMD phenotype in a mouse model of the disease using the genome editing technology. And in early 2016, he authored one of three papers published simultaneously in Science on fixing DMD in mice.
In 2015, researchers from Toronto's Hospital for Sick Children corrected an exon duplication in cells taken out of a patient with DMD, leading to production of regular dystrophin.
Following these early successes, funding has risen for CRISPR research in DMD. For example, last July, the University of California, Los Angeles received $2.2 million from the California Institute of Regenerative Medicine to evaluate the feasibility of CRISPR DMD gene therapy.