Farah Sheikh, PhD, on Developing Gene Therapy for Arrhythmogenic Right Ventricular Cardiomyopathy

“Oftentimes, people think a single gene is really not enough to bring the whole thing back together, because it's on its own. But what we found is that it actually acts as a magnet for all of these other proteins to sort of come back to that location and sort of prevent the cells from dying. And then obviously, that then prevents the structural damage, and also the scarring that would occur if you don't get cells being lost.I think this is an innovative finding, because everyone always thinks you have to add all of these different genes back together to get this complex to sort of form and anchor back together, and we showed that a single gene is able to do that particular function.”

Farah Sheikh, PhD, professor of medicine, University of California and her lab recently developed a mouse model of arrhythmogenic right ventricular cardiomyopathy (ARVC) that better models the disease phenotype in humans. The lab is also evaluating the novel LX2020 gene therapy in these models.

Sheikh presented the preclinical data at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California. CGTLive spoke with Sheikh to learn more about the preclinical data from the LX2020 gene therapy, benefits of the new mouse model her lab developed, and further research that remains to be done and questions that remain to be answered with ARVC.

Click here to read more coverage of the ASGCT 2023 meeting.

REFERENCE
Sheikh F, Zhang J, Nair A, et al. LX2020-An AAV based gene therapy improves the arrhythmogenic right ventricular cardiomyopathy phenotype in a severe mouse model harboring human PKP2 mutation. Presented at: ASGCT 2023 Annual Meeting; May 16-30; Los Angeles, California. Abstract #205