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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.
7 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.
9 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.
7 hours ago
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.
9 hours ago
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 ago
Two Years In, Elevidys Shows Functional Benefit in Older Boys With Duchenne
Boys with Duchenne who received Elevidys between ages 8 and 12 kept more motor function over two years than matched untreated boys, Sarepta reported at the World Muscle Society Congress. Here is what the data show, and what they don't.
2 days ago
No, CRISPR Hasn't Cured Down Syndrome. Here's What the Viral Study Really Showed
A viral post claims scientists can now remove the extra chromosome behind Down syndrome. The 2025 Mie University study is real, but it was done in lab-grown cells, not babies, and it is far from a treatment. Here is the myth vs. fact.
5 days ago
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