A complement C5-targeted GalNAc-conjugated siRNA with sustained efficacy in a non-human primate model of IgA nephropathy

By: Xueyan Zhang · Xue Zhang · Qiuyu Yang · Bin Kang · Mo Dan · Chunlei Li · Yan Chen · Jianhua Gan · Xiaoye Su

13 August 2026 at 08:00

This study characterizes a GalNAc-C5 small interfering RNA with potent in vitro and in vivo activity. Single subcutaneous dosing sustains long-term C5 suppression in cynomolgus monkeys with IgA nephropathy, outperforming Nefecon in blocking glomerular complement deposition, supporting its standalone or combinational clinical application.

In vivo-directed evolution identifies AAV-WM04 as a next-generation vector for potent and sustained hearing restoration in DFNB9

Molecular Therapy

By: Yong Tao · Cenfeng Chu · Zhenzhe Cheng · Shan Yin · Yilin Sun · Ying Chen · Huifang Zhang · Shuyue Bao · Boyu Yang · Baoyi Feng · Xianyu Huang · Yao Lu · Qiuxiang Yang · Xuegao Mao · Qifang Zhou · Chenxi Jin · Zhuo Duan · Guisheng Zhong · Hao Wu

13 July 2026 at 08:00

AAV-WM04, an AAV vector identified through in-vivo-directed screening in the adult cochlea, enables highly efficient and selective inner hair cell transduction. Dual-AAV delivery of OTOF using AAV-WM04 restores hearing in a DFNA9 deafness mouse model at low doses, highlighting its translational potential for gene therapy.

Integrative Multi-Omics Mendelian Randomization Analysis Identifies NIT2 as a Potential Metabolic Risk Gene in Hepatocellular Carcinoma

Omics in Hepatocellular

By: Ziyue Liao · Qinghua Fang · Yue Chen · Xiangyu Yang · Haijuan Wu · Hongyang Wang · Jian Gao

2 September 2026 at 18:00

J Gene Med. 2026 Sep;28(9):e70111. doi: 10.1002/jgm.70111.

ABSTRACT

BACKGROUND: Metabolic pathways are crucial in hepatocellular carcinoma (HCC) pathogenesis, but causal metabolic genes remain unclear. This study used Summary data-based Mendelian Randomization (SMR) and colocalization to identify metabolism-related genetic loci influencing HCC risk.

METHODS: Differentially expressed genes in hepatic malignancy phenotype versus normal tissues from TCGA and GTEx were analyzed. Metabolism-related candidates were examined via SMR and colocalization using multi-omics data: methylation (mQTL), expression (eQTL), and protein (pQTL) quantitative trait loci.

RESULTS: Multi-omics integration identified NIT2 as a key metabolic regulator for HCC. The cg13016775 locus of NIT2 was associated with elevated HCC risk at gene (OR = 1.618, 95% CI: 1.199-2.182) and protein (OR = 4.432, 95% CI: 1.783-11.018) levels. Colocalization supported a shared causal variant (PPH4 > 0.6), linking NIT2 to hepatocarcinogenesis via metabolic regulation.

CONCLUSIONS: This study provides multi-omics evidence for NIT2 as a potential causal gene in HCC, enhancing understanding of metabolic contributions to HCC pathogenesis and highlighting integrative genomics for uncovering causal relationships.

PMID:42681890 | PMC:PMC13534973 | DOI:10.1002/jgm.70111

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