Collectively, the data from in vitro and in vivo studies using FUS, TDP-43, and C9orf72 models demonstrate that hUPF1 holds significant potential for treating the majority of patients with ALS and ...

GI Innovation, a South Korean biotech company, announced on May 13 that it has been selected as a semi-finalist in the XPRIZE ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...

This technique streamlines quality control in gene therapy vector production, potentially accelerating manufacturing and improving clinical outcomes. This summary was automatically generated using ...

One of these lead assets is LX2006, an AAV-based gene therapy designed to treat patients with Friedreich's ataxia hypertrophic cardiomyopathy, by increasing expression of the heart muscle protein ...

The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an ...

Sarepta's gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the dystrophin protein that is deficient in patients with the muscle-wasting disease.