In a 13-year follow-up by St. Jude Children’s Research Hospital and University College London published in The New England ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages.

The world’s first patient to successfully receive a CRISPR gene-editing treatment was discharged from Children’s Hospital of Philadelphia on June 2, ABC News reported.  KJ Muldoon, a 10-month-old who ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

“The promise of gene therapy that we’ve heard about ... from celebrity news to compelling human interest stories. With their success, KJ’s parents are now thrilled to have their son home ...

CRSP aims to extend its gene-editing lead with in vivo candidates after Casgevy's ex vivo breakthrough success.

The success story represents one of the first documented ... at CHOP and Dr. Kiran Musunuru at Penn, decided to apply gene therapy. Within six months, they had devised and manufactured a treatment ...

CureLab Veterinary Inc., a pioneer in developing innovative veterinary therapeutics to combat cancer and inflammatory ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...