After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...
Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...
Using prime editing, NCF1 mutation is corrected in a 19-year-old with chronic granulomatous disease, an inherited immune disorder, marking a gene therapy milestone.
A groundbreaking Cleveland Clinic trial is demonstrating how gene-editing technology could transform the way millions of Americans manage high cholesterol. A Phase 1 clinical trial at Cleveland Clinic ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing mutations. The technology could advance treatments for cystic fibrosis and ...
Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common ...
Bismuthene nanodiscs use mild heat to switch on gene editing that dismantles a tumor's heat defenses, creating a feedback loop that kills cancer cells more effectively.
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