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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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.
3 days 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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GENE TECH TIMES
The Future Has Arrived
Gene Therapy Successfully Reverses Heart Failure in Minipig Study: A Big Step in Heart Failure Treatment
Heart failure, which affects over 64 million people worldwide, is currently considered irreversible without a heart transplant. Researchers at the University of Utah have developed a gene therapy that could change this. Using minipigs as a model, scientists successfully reversed heart failure by delivering a therapeutic gene called cBIN1 directly into heart cells. The therapy, which targets a key protein often deficient in heart failure patients, resulted in significant recov
Dec 11, 2024
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