Farah Sheikh, PhD, on Modeling Arrhythmogenic Right Ventricular Cardiomyopathy

“One of the big things that our lab really does is to model cardiomyopathy disease in mice, and trying to find an appropriate model, not only to look at severe forms, but also milder forms of the disease. So, the presentation really entails talking about this new mouse model that we generated, to better understand the disease progression, which basically uses PKB2 as a sort of model.”

Researchers from the University of California – San Diego in the lab of Farah Sheikh, PhD, professor of medicine, have developed a mouse model of arrhythmogenic right ventricular cardiomyopathy (ARVC) that better models the disease phenotype in humans and have evaluated the novel LX2020 gene therapy in these models.

Data from the research were presented at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California, by Sheikh. CGTLive spoke with Sheikh to learn more about the mouse model that her lab developed and how it improves upon currently used mouse models. She discussed how her lab saw benefits not only with giving the gene therapy preventatively or early on in the ARVC disease course but also later on in the disease, similarly to how humans would likely be treated. She noted other findings observing the gene therapy in mouse models, such as improving right ventricular function and preventing arrythmias.

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