A biomedical researcher at the Southwestern Medical Center at the University of Texas has been awarded a grant of $250,000 for a study of the potential for the use of CRISPR-Cas9 Duchenne Muscular Dystrophy treatment in humans.
CRISPR-Cas9 is a novel gene editing technique that can be used to alter DNA with precision. With the CRISPR-Cas9 system it is possible to add, remove, or alter sections of DNA associated with a wide range of genetic diseases.
While there is considerable excitement about the technique – which is easier that other gene editing methods – questions have been raised about its safety. While it is possible to edit very specific sections of DNA using guide RNA to bind to certain genes and Cas9 enzymes to make the cuts, there is concern about unintentional gene edits. One trial on human subjects was performed last year by Chinese biomedical researchers, although to date the technique has not been used in human clinical trials in the United States.
Previous studies on mdx mice have shown CRISPR-Cas9 has great potential for the treatment of Duchenne muscular dystrophy. Last year, mdx mouse studies demonstrated CRISPR-Cas9 could be safely used to edit DMD genes, with the mice showing histological and functional improvements. Trials on humans have yet to be conducted.
Due to the potential of the technique for the treatment of Duchenne muscular dystrophy, funding has now been awarded to thoroughly examine the safety of the technique. Eric Olson, PhD., of UT Southwestern’s Department of Molecular Biology will lead the study with the funding provided by Parent Project Muscular Dystrophy through the Gene Transfer Initiative.
The study will look at any risks associated with the use of CRISPR-Cas9 treatment of Duchenne muscular dystrophy in humans, including any immune responses to the AAV9 used to perform the gene edits. Olson and his team of researchers will look for any unintentional gene edits and the effects of those unintentional cuts.
Dystrophin proteins synthesized following CRISPR-Cas9 treatment will be compared with those synthesized following other gene editing techniques. In addition to assessing safety, the researchers hope to gain a much better understanding how the CRISPR-Cas9 system works.
Olson said, “We are optimistic about the potential of CRISPR-Cas9 to help Duchenne patients. There is still work to do, and this grant will help maintain the momentum of our research activities.”