AAV gene replacement restores Parkin pathway activity in preclinical tests
An AAV carrying functional human PRKN restored mitochondrial-pathway signaling in Parkin-deficient cells and patient fibroblasts, while rodent tests showed that ...
Tag: AAV gene therapy
AAV RNAi Targeting Ataxin-2 Extends TDP-43 Mouse Survival
In an exciting breakthrough that could revolutionize therapeutic approaches for neurodegenerative diseases, a team of scientists has unveiled a novel ...
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