#gene therapy — Wednesday, September 16, 2026 (2 items)
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Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.
(nature.com)
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Nature Medicine 2026-09-16 | #gene therapy #hemophilia #aav #coagulation | |
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Structure-guided discovery and engineering of miniature CRISPR-Cas12m for epigenome editing.
(nature.com)
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Nature Structural & Molecular Biology 2026-09-16 | #crispr #epigenomics #protein design #structural biology #gene therapy |