A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...
Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common illnesses. So far, only 15 people have received the treatment. But, if all ...
In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...
A single infusion of an experimental gene-editing drug appears safe and effective for cutting cholesterol, possibly for life, according to a small early study released Saturday. The study, which ...
Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...
The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex ...
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