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RESEARCH / LITERATURE

Research Literature

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In Vivo Cell Engineering & Gene Editing 5

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

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Original research
Nature Materials

In situ CAR engineering and nanoparticle-mediated TREM2 degradation for macrophage reprogramming in malignant ascites

Yijia Li; Ting Yang; Xiyue Zhang; Rui Peng; Jifang Gong; Zihan Zhang; Bin Ma; Yu Hou; Yunxuan Liu; Kemeng Chen; Qidong Bai; Yian Fang; Changdi Cao; Jia Hao; Zhiwei Sun; Ziqiong Jiang; Mengying Dong; Chenlong Wei; Zhongtang Li; Qiang Zhang; Lin Shen; Xueqing Wang; Mi Deng; Changsong Qi; Lei Miao

A Nature Materials study describes mRNA-loaded LNPs that reprogram tumor-associated macrophages into Claudin18.2-targeting CAR macrophages. The lipid formulation also promotes TREM2 internalization and degradation, reshaping immune responses. The original abstract reports tumor suppression and longer survival with PD-1 blockade in mice, plus ex vivo patient-sample findings. These findings do not demonstrate clinical efficacy in treated patients.

mRNA / LNPIn Vivo Cell Engineering & Gene EditingProtein degradation / PROTAC
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DOI: 10.1038/s41563-026-02769-2

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

Original research
Science

CAR T cells produced in vivo to treat cardiac injury

Joel G Rurik; István Tombácz; Amir Yadegari; Pedro O Méndez Fernández; Swapnil V Shewale; Li Li; Toru Kimura; Ousamah Younoss Soliman; Tyler E Papp; Ying K Tam; Barbara L Mui; Steven M Albelda; Ellen Puré; Carl H June; Haig Aghajanian; Drew Weissman; Hamideh Parhiz; Jonathan A Epstein

The 2022 study delivered CAR-encoding mRNA with CD5-targeted lipid nanoparticles, generating transient CAR T cells in mice. The abstract reports reduced cardiac fibrosis and improved function after injury. This is preclinical evidence, not human efficacy.

In Vivo Cell Engineering & Gene EditingmRNA / LNP
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DOI: 10.1126/science.abm0594

Original research
Nature Nanotechnology

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

Qiang Cheng; Tuo Wei; Lukas Farbiak; Lindsay T Johnson; Sean A Dilliard; Daniel J Siegwart

This 2020 study added a SORT component to lipid nanoparticles to alter tissue targeting and deliver mRNA or CRISPR cargoes. It provides preclinical evidence for extrahepatic delivery, rather than proof of human tissue targeting or clinical benefit.

mRNA / LNPIn Vivo Cell Engineering & Gene Editing
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DOI: 10.1038/s41565-020-0669-6

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