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 ...

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

More information: Maria Gioulvanidou et al, Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy, Human Gene Therapy (2024). DOI: 10.1089/hum.2024.165 Provided by Mary Ann Liebert, Inc ...

Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy.

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI ...

Jayden Wilsey, 15, of Falmouth, is undergoing human gene therapy for sickle cell disease at Boston Children's Hospital. The hospital's sickle cell program currently has 450 patients under the age ...

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 ...