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Polygence Scholar2024
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Prisha Pachpande

Class of 2025Troy, Michigan



  • "How can gene editing therapies lead to the diagnosis and treatment of Leber Congenital Amaurosis?" with mentor Rebecca (May 27, 2024)

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

Started Apr. 14, 2023

Abstract or project description

The recent utilization of RNA-based antisense oligonucleotide therapy, CRISPR/CAS9, ZFN, TALEN, gene augmentation therapy and many more technologies to help edit genome sequences has proven to be revolutionary in the scientific field. More specifically, these editing strategies has been tested with a particularly unique disease, LCA. 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 such as the RPE65 Gene and the LCA5 gene are known to be causative of LCA. First, I will Introduce LCA, and what symptoms it comes with, as well as what genes it mainly targets. I will then review primary literature on various gene editing therapies and how they are currently being applied, and examples of already tested diseases. 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. Lastly, I propose CRISPR and TALEN would provide the most therapeutic benefit to patients with LCA.