Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice

AAV-mediated FGF21 gene therapy extends health span and lifespan in aged mice through multi-tissue adaptations

This study demonstrates that a one-time muscle-directed AAV-mediated FGF21 gene therapy in aged and geriatric mice extends health span and l

FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program

The FDA has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy for trea

FDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorder

The FDA has cleared an Investigational New Drug (IND) application for MMA-101, an AAV-based gene therapy candidate developed with NIH suppor

Gene therapy restored hearing in deaf patients

Life Biosciences Conducts First Gene Therapy Trial to Reverse Cellular Aging in Patient with Glaucoma

Life Biosciences, a Boston-based biotech startup, has conducted the world's first gene therapy on a living patient aimed at reversing cellul

Novel Blood-Brain Barrier Modulation Strategy Shows Promise for Alzheimer's Treatment Through Enhanced Amyloid-β Clearance

This scientific research presents a novel therapeutic approach for Alzheimer's disease that targets the blood-brain barrier (BBB) to enhance