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Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial

Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04636-8

In this single-arm phase 1 trial, an AAV gene therapy carrying the Padua variant of factor IX was well tolerated in 11 adolescents with hemophilia B and led to reductions in annualized bleeding rate.
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Hypoxia-induced XBP1s-MYDGF axis suppresses ferroptosis through UBQLN1-mediated stabilization of LCN2 in gastric cancer

Oncogene, Published online: 07 April 2026; doi:10.1038/s41388-026-03760-6

Hypoxia-induced XBP1s-MYDGF axis suppresses ferroptosis through UBQLN1-mediated stabilization of LCN2 in gastric cancer
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Unified modeling of 3D molecular generation via atomic interactions with PocketXMol

A versatile, atom-level generative AI model enables unified pocket-interacting tasks, from docking to de novo design, and demonstrates robust experimental validation for both small-molecule and peptide therapeutics.
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Dual function of DOT1L suppresses tumor cell-intrinsic immunogenicity in hepatocellular carcinoma

Oncogene, Published online: 31 March 2026; doi:10.1038/s41388-026-03744-6

Dual function of DOT1L suppresses tumor cell-intrinsic immunogenicity in hepatocellular carcinoma
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