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Prime Medicine in vivo prime editing clinical trials
Overview
Prime Medicine has initiated a Phase 1/2 clinical trial using in vivo prime editing to treat Wilson disease, a rare genetic condition caused by mutations in the ATP7B gene. This method utilizes lipid nanoparticles to deliver editing machinery directly to liver cells to correct mutations, aiming for a permanent, “one-and-done” solution rather than lifelong medication.
This approach represents a shift in gene therapy, moving from traditional methods that require modifying cells outside the body to a “search-and-replace” engine capable of rewriting DNA directly inside the human body with minimal genomic damage. While the current focus is on Wilson disease, researchers suggest this technology could eventually be applied to common conditions such as heart disease, obesity, and diabetes.
Entities
Prime Medicine · David R. Liu · University of Michigan Health Frankel Cardiovascular Center · Wilson disease · Nature
Timeline
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[TECHNOLOGY] 3 sourcesGene editing technology advances toward treating common diseases
Gene editing is expanding from rare disease treatments to common conditions like heart disease, with new prime editing trials testing in-body DNA rewriting for Wilson disease.
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[HEALTH] 10 sourcesPrime Medicine treats first patient with in-body DNA prime editing
Scientists have used prime editing to correct a Wilson disease mutation directly inside a human body for the first time, potentially offering a one-time permanent cure via lipid nanoparticles.
Sources
90minutos.co · cumhuriyet.com.tr · ekriti.gr · exaudi.org · goodmenproject.com · kibrismanset.com · koganpage.com · lomazoma.com · odelalis.gr · raillynews.com · singularityhub.com · theibulletin.com · upday.com