#gene therapy — Sunday, March 01, 2026 (3 items)
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In vivo base editing of Chd3 rescues behavioural abnormalities in mice.
(nature.com)
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Nature 2026-03-01 | #gene therapy #aav #neurodevelopmental #cognitive | |
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Circular RNA aptamers targeting neuroinflammation ameliorate Alzheimer disease phenotypes in mouse models.
(nature.com)
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Nature Biotechnology 2026-03-01 | #neurodegeneration #gene therapy #neuroprotection #neuroinflammation #aptamers | |
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In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial.
(nature.com)
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Nature Medicine 2026-03-01 | #clinical trial #gene therapy #atherosclerosis #hypercholesterolemia |