Prisha Pachpande

How can gene editing therapies lead to the diagnosis and treatment of Leber Congenital Amaurosis?

Started Apr. 14, 2023

Abstract or project description

Leber Congenital Amaurosis (LCA), a genetic condition, is a rare eye disease that affects the retina, causing blindness. The clinical trials for the treatment have so far been unsuccessful, so there is no determined cure. [Mutations in genes (xyz) are known to be causative of LCA]. I will review primary literature on various gene editing therapies and how they are currently being applied. I will then propose how these potential technologies could be utilized in the treatment and care of this disease. Afterwards, I will run through the different pros and cons that these therapies could pose, and which one is most suitable for further action. I propose [strategy abc], would provide the most therapeutic benefit to patients with LCA.