Breakthrough Study Supports Genetic Editing as a Potential Treatment for Rare Rapid-Aging Disease Progeria
Retrieved on:
Wednesday, January 6, 2021
Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks.
Key Points:
- Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks.
- Zokinvy increases average lifespan by 2.5 years and improves some symptoms of Progeria, but it does not address the genetic mutation that causes the disease.
- Using a newer form of genetic editing, known as base editing, researchers found that they could reverse the mutation that causes Progeria, improving disease symptoms and dramatically increasing lifespan in a Progeria mouse model.
- The Progeria Research Foundation (PRF) is a nonprofit organization established in 1999 by the family of Sam Berns, a child with Progeria.