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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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
Regenerating Hearts: How Stem Cell Therapy Could Transform the Treatment of Congenital Heart Diseases
Heart disease is a leading cause of death worldwide, affecting millions at every stage of life, including infants born with congenital heart defects. These conditions often result in a weakened heart, leading to symptoms such as chest pain, breathlessness, and swelling. If untreated, they can be fatal. While surgeries can offer temporary relief, many patients eventually face heart failure. They may require a heart transplant—a challenging solution due to the scarcity of donor
Nov 30, 2024
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