What happens when the mutation causing a disease is also key to unlocking its treatment? Beghou Partner Vishal Singal explores the clinical and patient perspectives on retinitis pigmentosa and the outlook for cell & gene therapies in ophthalmology with guests: Dr. Marianna Weener, Retinal Physician Scientist at the Broad Institute of MIT and Harvard Lulia Kim, an individual living with retinitis pigmentosa Hear the following takeaways: How a repetitive mutation could be a “founder effect,” and how such genetic variants may form the basis for treatment Why gene editing, rather than gene replacement, may be the preferred treatment tool in an ophthalmologic disease state The gap between the kinds of endpoints currently required by regulators versus those that are more realistic for patients with slowly progressing retinal disease
