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