top of page
LATEST NEWS
Optogenetic Gene Therapy Gives Some Blind Patients Back a Sense of Light
In the NEJM-published PIONEER trial, an algae-derived light-sensing gene plus amber-light goggles improved light sensitivity in 6 of 10 people with late-stage retinitis pigmentosa, and 4 of 8 got better at finding objects. Here is how optogenetics works.
9 hours ago5 min read
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.
1 day 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.
1 day ago5 min read
LEARN MORE
LEARN MORE
GENE TECH TIMES
The Future Has Arrived
Optogenetic Gene Therapy Gives Some Blind Patients Back a Sense of Light
In the NEJM-published PIONEER trial, an algae-derived light-sensing gene plus amber-light goggles improved light sensitivity in 6 of 10 people with late-stage retinitis pigmentosa, and 4 of 8 got better at finding objects. Here is how optogenetics works.
9 hours ago
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.
1 day 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.
1 day 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.
2 days 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.
3 days ago
bottom of page