A single infusion of adeno-associated virus (AAV)-mediated gene therapy led to sustained clinical benefit and no late-onset ...

Researchers say they've found a promising new way to cure certain blood disorders using gene therapy. Trump Administration ...

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

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

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

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 ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...