#gene therapy — Thursday, July 30, 2026 (2 items)
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Gene therapy for hypertrophic cardiomyopathy: From feasibility to a fieldwide framework.
(linkinghub.elsevier.com)
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Cell Med 2026-07-30 | #gene therapy #cardiomyopathy #vector #immunosuppression #clinical translation | |
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AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne Syndrome.
(jci.org)
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Journal of Clinical Investigation 2026-07-30 | #gene therapy #cockayne-syndrome #neurodegeneration #aav #dna repair |