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PEABODY, Mass., Jan. 6, 2021 /PRNewswire/
The Progeria Research Foundation (PRF) announced today the publication of a breakthrough preclinical mouse study in the journal
Nature, demonstrating the substantial effect of genetic editing in directly reversing the mutation that causes Hutchinson-Gilford Progeria Syndrome (HGPS, or Progeria). Scientists at Broad Institute, the National Institutes of Health (NIH), PRF and others contributed to this ground-breaking study, indicating a critical new pathway toward correcting the root cause of Progeria.
Progeria is an ultra-rare, fatal, pediatric rapid-aging disease, affecting approximately 400 children worldwide. 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