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LATEST NEWS
FDA Proposes One Approval for Many Mutations in Personalized Gene Editing
A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.
3d4 min read
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.
Oct 224 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.
Oct 224 min read
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The Future Has Arrived
Mini-Brain Study Uncovers Key Genetic Link to Autism Spectrum Disorder
In a promising study, scientists from the Mayo Clinic and Yale University utilized brain organoids—tiny brain models grown from human cells—to investigate the link between autism spectrum disorder (ASD) and the specific brain cell imbalances associated with the condition. The researchers discovered that individuals with ASD exhibit a notable imbalance in excitatory cortical neurons in the forebrain, which varies depending on the individual's head size. This finding suggests t
Apr 30, 2024
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