People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

Gene therapy, the idea of fixing faulty genes ... with a specific form of inherited deafness called DFNA50 caused by mutations in microRNA, by using a novel in vivo CRISPR genome editing ...

An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life ...

In this Revvity GENCast episode 2, experts will discuss applications of gene editing to improve scientists’ understanding of the relationship between variant and disease in functional genomics studies ...

Marley Mansour, 11, is one of fewer than 100 people in the world with a particular mutation in the NARS1 gene. She's set to ...

Fundraising efforts have helped jump-start gene therapy that could help patients regardless of mutation. While it likely won't be available for years, "just to have these therapies in trials ...