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Breakthrough Study Supports Genetic Editing as a Potential Treatment for Rare Rapid-Aging Disease Progeria


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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 do ....

United Kingdom , David Liu , Francis Collins , Sam Berns , Leslie Gordon , Jonathan Brown , Division Of Cardiovascular Medicine At Vanderbilt University , Broad Institute , Merkin Institute Of Transformative Technologies In Healthcare , National Human Genome Research Institute , International Progeria Patient Registry , Progeria Research Foundation , Richard Merkin Professor , Merkin Institute , Transformative Technologies , Senior Investigator , Assistant Professor , Cardiovascular Medicine , Progeria Patient Registry , ஒன்றுபட்டது கிஂக்டம் , டேவிட் லியூ , பிரான்சிஸ் கோலின்ஸ் , சாம் பெர்ன்ஸ் , லெஸ்லி கோர்டந் , ஜொனாதன் பழுப்பு , பிரிவு ஆஃப் இருதய மருந்து இல் வாண்டர்பில்ட் பல்கலைக்கழகம் ,