HACKENSACK, N.J., April 8, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), awarded the University of Missouri School of Medicine a bridge grant for $31,500 to continue evaluating CRISPR therapy in a pre-clinical model of Duchenne. The project is led by Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.
Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the DMD gene that codes for the dystrophin protein. Currently, a number of strategies are in development to explore the possibility of using gene editing to restore production of a functional dystrophin protein.
Gene editing utilizing AAV-mediated CRISPR/Cas9 is an area of therapeutic development that has significant potential for treating individuals with Duchenne. The use of the CRISPR/Cas9 technology in Duchenne is still in early stages, with various strategies being investigated for how best to modify the DMD gene to restore production of the dystrophin protein. While there is excitement in the potential that gene editing holds, the Duchenne community recognizes that there are many questions that still remain unanswered.
One such question is around the topic of safety. Specifically, if the Cas9 enzyme, which is a bacterial protein, will cause an immune response when it is delivered throughout the body to reach the muscle. Dr. Duan will continue his ongoing investigation into this question by exploring immune responses from systemic delivery of AAV-mediated CRISPR/Cas9 in a large pre-clinical model.
"Duchenne research has progressed significantly over the past several years, with strategies such as gene editing holding incredible possibilities for our community," said Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation. "The work Dr. Duan is doing will contribute greatly to our understanding of how we can safely translate CRISPR/Cas9 gene editing strategies to patients with Duchenne."
"We greatly appreciate the support from PPMD and the Duchenne community," said Dr. Duan. "CRISPR/Cas9 editing therapy has the potential to permanently repair the mutated DMD gene. Studies from many groups, including us, have demonstrated efficient restoration of dystrophin in patient cells and rodent models. Yet, little is known about the immune response to the bacterial derived Cas9 protein. A better understanding on Cas9 immunity will pave the way to the translation of this promising therapeutic modality."
To learn more about PPMD's robust Research Strategy, funding initiatives and strategies for accelerating drug development, click here.
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals.
Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.