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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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.
1 day ago4 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
Scientists Cure Type 1 Diabetes in Mice Using a Gentle Immune System “Reset”
Stanford Medicine researchers cured Type 1 diabetes in mice using a gentle immune-reset approach that combines donor blood stem cells and pancreatic islet transplants. The method prevented autoimmunity, required no immunosuppressive drugs, and may pave the way for future treatments for diabetes and other autoimmune diseases.
Dec 2, 2025
Enhance CAR T-Cell Therapy Efficacy and Reduce Toxicity by Using the Microbiome
Emerging research shows that the intestinal microbiome plays a vital role in the success of CAR T-cell therapy, influencing both efficacy and toxicity. Dr. Melody Smith’s studies reveal that certain antibiotics can disrupt the microbiome, reducing overall survival and increasing neurotoxicity risks. Optimizing gut health through diet or microbiome-targeted interventions may enhance CAR T-cell outcomes for cancer patients.
Feb 24, 2025
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