Despite emerging clinical successes, current genome editors suffer from off-target effects and can trigger unwanted responses from the immune system, limiting their broader therapeutic applications.

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 ...

The 2025 International Summit on Human Genome Editing called for: • A moratorium on heritable edits until safety and societal consensus are achieved Meanwhile, patients with devastating genetic ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...