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AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids.

作者信息

Gupta Navin, Zhang Ke, Sabbisetti Venkata, Shu Jian, Morizane Ryuji

机构信息

Nephrology Division, Department of Medicine, Mass General Brigham, Harvard Medical School, Boston, MA, USA.

Cutaneous Biology Research Center, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

出版信息

Signal Transduct Target Ther. 2025 Aug 8;10(1):252. doi: 10.1038/s41392-025-02336-2.


DOI:10.1038/s41392-025-02336-2
PMID:40774951
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12332185/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7175/12332185/73f929f87330/41392_2025_2336_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7175/12332185/73f929f87330/41392_2025_2336_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7175/12332185/73f929f87330/41392_2025_2336_Fig1_HTML.jpg

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本文引用的文献

[1]
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies.

Mol Ther Methods Clin Dev. 2024-5-29

[2]
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis.

Front Immunol. 2022

[3]
Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair.

Sci Transl Med. 2022-3-2

[4]
Three-dimensional intact-tissue sequencing of single-cell transcriptional states.

Science. 2018-6-21

[5]
Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.

Yale J Biol Med. 2017-12-19

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