#gene therapy — Tuesday, April 21, 2026 (2 items)
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Uncovering mitochondrial defects in photoreceptors opens therapeutic opportunities for Stargardt disease.
(pnas.org)
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PNAS 2026-04-21 | #mitochondrial #gene therapy #microrna #retinal degeneration | |
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Human promoter-driven AAV tools enable precision gene therapy targeting cochlear hair cells.
(linkinghub.elsevier.com)
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Cell Reports Medicine 2026-04-21 | #gene therapy #aav #targeted delivery #hearing loss #promoter |