The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely rare genetic diseases. Doctors say they constructed a bespoke gene ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. The baby, KJ Muldoon of Clifton Heights ...

You can reach Jason on Signal at JasonMast.05. NEW ORLEANS — The mood at the annual meeting of the American Society of Cell and Gene Therapy isn’t amazing. The biotech market has been bad for ...

A flexible, intrinsically disordered region (IDR) in the TAF2 protein directs its movement between gene promoters and nuclear speckles, linking transcription initiation and RNA processing.

For more than half a century, KISS' Gene Simmons has been one of the most recognizable figures of rock and roll. Simmons rose to stardom as the "demonic" bassist and co-lead singer of the rock ...

A physician reflects on the burden of caregivers and the tragic story of Gene Hackman and Betsy Arakawa. He also emphasizes the importance of community support for caregivers and encourages acts ...

Gene therapies, which can be extremely expensive to develop, generally target more common disorders in part for simple financial reasons: more patients mean potentially more sales, which can help ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

You can reach Megan on Signal at mmolteni.13. Leading trade organizations representing the makers of cell and gene therapies are calling for a 10-year international moratorium on the use of CRISPR ...