Silence‑and‑Replace Gene Therapy Stops and Reverses SPG4 Hereditary Spastic Paraplegia in Mice

Search.

Breaking: “Silence‑adn‑Replace” Gene Therapy Stops Hereditary Spastic Paraplegia in Mice

December 15 2025 – Researchers from Drexel University College of Medicine and UMass Chan Medical School announced a proof‑of‑concept that a single viral‑vector gene‑therapy can prevent and even reverse the hallmark gait defects of hereditary spastic paraplegia (HSP) in a mouse model.

What the Study Shows

A specially engineered adeno‑associated virus (AAV9) delivers micro‑RNA to silence the mutant SPAST gene and simultaneously supplies