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Research Literature

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In Vivo Cell Engineering & Gene Editing 2

Duplicate DOIs are merged. This is a selected collection, not an exhaustive bibliography.

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Original research
Nature

Cancer-selective frameshifting circuits show preclinical activity with systemic AAV9 delivery

Qiuyi Wang; Ao Chen; Yaoqi Liu; Xiaoxiao Yu; Hao Zhang; Yuqiang Yu; Pengyu Chen; Yue Zhang; Francis Ka-Ming Chan; Xiangwei Gao; Yuanhui Mao

A Nature study describes FAST gene circuits that exploit cancer-associated ribosomal frameshifting to conditionally express gasdermin D and trigger pyroptosis. According to the original abstract, systemic AAV9 delivery produced tumor regression and immune activation in mouse colorectal and pancreatic cancer models, with synergy alongside checkpoint blockade. These are cell and animal findings, not human clinical evidence.

In Vivo Cell Engineering & Gene Editing
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DOI: 10.1038/s41586-026-11200-4

Original research
Nature Materials

An inherent T cell-activating mRNA delivery carrier for in vivo CAR T generation

Qiannan Cao; Yingli Yao; Wenming Zheng; Hongqian Liu; Mingxia Jiang; Dayang Xie; Siting Zhang; Pijun Su; Huilin Yuan; Xiaoyuan Chen; Huapan Fang; Huayu Tian

A polymer-lipid carrier incorporating arginine-modified oligoethylenimine combined T-cell activation with mRNA delivery without an added targeting ligand. The formulation favored splenic delivery and engaged PI3K-AKT-mTOR signaling. FAP-CAR mRNA generated functional CAR T cells in experimental cancer and fibrosis settings. These are preclinical results; the article also has a subsequent publisher correction that should accompany interpretation.

In Vivo Cell Engineering & Gene EditingmRNA / LNP
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DOI: 10.1038/s41563-026-02675-7

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