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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.
24 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.
2 days 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.
2 days ago5 min read
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GENE TECH TIMES
The Future Has Arrived
Five-Year Follow-Up Shows Sustained Visual Improvements in LHON Patients Treated with AAV Gene Therapy
A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide long-lasting visual improvements for patients with Leber hereditary optic neuropathy (LHON). This genetic condition, caused by a mutation in the MT-ND4 gene, typically leads to severe vision loss. The study followed patients who received the therapy and found significant improvements in vision even five years after treatment. The therapy involv
Dec 27, 2024
Serious Immune Reaction in a Young Patient Challenges Rett Syndrome Clinical Trial
A clinical trial for NGN-401, a gene therapy designed to treat Rett syndrome, recently faced a setback when a young participant experienced a severe immune reaction after receiving a high dose of the treatment. This reaction, called systemic hyperinflammatory syndrome, is a rare and dangerous immune response, causing her condition to become critical. As a result, high-dose treatments have been paused. Rett syndrome is a genetic disorder that primarily affects girls, leading t
Nov 22, 2024
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