Recent advances in artificial intelligence are accelerating breakthroughs in genomics, from predicting disease-causing mutations to engineering proteins and modeling brain disorders. Tools like Evo 2, ...

Ocugen's gene-agnostic therapy strategy targets broad retinal patient groups with one-time treatments, setting up major 2026-27 catalyst events.

SYNGAP1 encephalopathy is a rare genetic disorder for which there is no treatment. It causes epilepsy, intellectual disability, psychomotor delay and, frequently, autism. It arises from mutations in ...

Researchers say a new AI system can identify disease-causing mutations and explain their biological effects, potentially ...

Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have ...

Researchers identified a new genetic mutation that may allow some people to survive, even thrive, on three hours of sleep. The mutation is called SIK3-N783Y. Scientists said mice test subjects ...

This photo provided by Emilys Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare genetic mutation, during a trip to Maine. (Emilys Entourage via AP) ...

A new gene therapy tested in China has improved the hearing of 38 people who were born deaf due to mutations in a gene called ...

The ability of different genetic variants—changes to one or more building blocks of DNA—to cause disease, and to what extent, ...

There are about 80,000 new cases of bladder cancer every year in the US alone. About one-third of cases are diagnosed at late stages when the disease is hard to treat. Scientists have now learned more ...

SYNGAP1 encephalopathy is a rare genetic disorder for which there is no treatment, causing epilepsy, intellectual disability, ...