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Prime Medicine Doses First Patient With an In Vivo Prime Editor for Wilson Disease
Prime Medicine has dosed the first patient with PM577a, a one-time prime-editing infusion designed to correct the H1069Q mutation in liver cells and restore copper excretion in Wilson disease. It is the company's first prime editor delivered inside the body.
8 hours ago5 min read


The 2026 Nobel Prize for Optogenetics Is Also a Gene Therapy Story
The 2026 Nobel Prize in Physiology or Medicine went to Karl Deisseroth, Peter Hegemann and Georg Nagel for light-gated ion channels and optogenetics. Here's how an algae gene became a tool for neuroscience, and why the first optogenetic gene therapy for blindness is now under FDA review.
11 hours ago5 min read


Cornell Scientists Build a Compact, Non-CRISPR Tool That Inserts Whole Stretches of DNA
Cornell researchers fused two bacterial proteins, TldR and TniQ, into a compact RNA-guided system that inserts large pieces of DNA at a chosen site without a CRISPR-Cas protein. It works only in bacteria so far, but it targets one of gene editing's biggest gaps.
1 day ago5 min read


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Prime Medicine Doses First Patient With an In Vivo Prime Editor for Wilson Disease
Prime Medicine has dosed the first patient with PM577a, a one-time prime-editing infusion designed to correct the H1069Q mutation in liver cells and restore copper excretion in Wilson disease. It is the company's first prime editor delivered inside the body.
8 hours ago
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