A small group of academics and government scientists, led by Dr. Francis Collins, is exploring gene editing as a potential highly effective treatment for progeria, a rare disease with no existing cure.
Gene editing techniques that have shown promise in mice are now being considered for a clinical trial aimed at slowing or halting the progression of progeria, a condition that affects very few individuals globally.
Dr. Kiran Musunuru, a gene editing researcher, highlighted the potential risks of transitioning gene editing from successful animal studies to human trials, emphasizing the uncertainty of efficacy.
Driven by a personal encounter with a progeria patient decades ago, Collins, now a prominent figure in the medical field, seeks to advance treatments for the ultrarare disease and improve the lives of individuals like Meg Casey.
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