Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression. This is a ...

Research presented by Dr Anna Keegan of University College London at the ELRIG meeting in Hinxton described how improved ...

Learn about gene expression studies in progressive supranuclear palsy in Mayo Clinic's Precision Medicine in Neurodegeneration Lab led by Nilufer Ertekin-Taner, M.D., Ph.D.

New research shows epigenetic silencing may limit durability of AAV gene therapy in haemophilia by reducing long term ...

Gene therapy has a reputation as a one-time intervention that has a lifelong effect. It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a ...

Gene and cell therapy is moving fast – and in a clear direction. Programs are pushing toward more complex payloads, virus-free engineering, and manufacturing models that can scale from early research ...