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LATEST NEWS
Estuary Launches With $103 Million to Bring Gene Therapy to Polycystic Kidney Disease
Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.
2 days ago4 min read


Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.
2 days ago4 min read


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.
Oct 115 min read


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Revolutionary Gene Therapy Targeting Galectin 1 Shows Promise in Reducing Liver Cancer Incidence in Animal Models
New Approach for Liver Cancer Treatment and Possible Prevention: Gene Therapy Targets Biomarker for Hepatocellular Carcinoma (HCC) Researchers at the UC Davis Comprehensive Cancer Center have unveiled a promising gene therapy approach that significantly reduces the incidence of hepatocellular carcinoma (HCC) in animal models, specifically mice. This innovative research targets a specific protein known as galectin 1 (Gal1), which has long been established as a critical biomark
Feb 2, 2024
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