Cas9 in germline editing presents opportunities for genetic disease correction, alongside risks of off-target effects and ...

Gene therapies are raising hopes for patients with rare disorders, various cancers, and complex neurological diseases. But there are ethical and policy challenges on the path from research to ...

Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart failure, powered by their proprietary human mini-Heart technology. FDA ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the peer-reviewed journal Human Gene Therapy. To improve the efficacy of gene therapy when ...

UC San Diego scientists have created a gene therapy that goes beyond masking Alzheimer’s symptoms—it may actually restore brain function. In mice, the treatment protected memory and altered ...

Medera Presented Updated Results from First-In-Human Gene Therapy Trial for Heart Failure with Preserved Ejection Fraction at 2025 HFpEF Summit Provided by GlobeNewswire Apr 1, 2025, 12:00:00 PM ...

Gene therapy gets a turbo boost Breakthrough technology could safely, efficiently treat hundreds of genetic diseases Date: August 29, 2024 Source: University of Hawaii at Manoa Summary: For ...

At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. However ...