#gene therapy — Wednesday, September 09, 2026 (2 items)
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Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy.
(science.org)
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Science Advances 2026-09-09 | #muscular dystrophy #suppressor trna #gene therapy #nonsense-mutations #aav | |
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Intravenous gene therapy improves life span and clinical outcomes in a feline model of Sandhoff disease.
(science.org)
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Science Translational Medicine 2026-09-09 | #gene therapy #lysosomal storage disease #neurodegeneration #aav vectors #animal-models |