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 ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects ...

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 ...

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 ...

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 ...

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 ...