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CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy.

作者信息

Gee Min Sung, Kwon Eunji, Song Myeong-Hoon, Jeon Seung Ho, Kim Namkwon, Lee Jong Kil, Koo Taeyoung

机构信息

College of Pharmacy, Kyung Hee University, Seoul, 02447, Republic of Korea.

Department of Biomedical and Pharmaceutical Sciences, Graudate School, Kyung Hee University, Seoul, 02447, Republic of Korea.

出版信息

Transl Neurodegener. 2024 Apr 12;13(1):21. doi: 10.1186/s40035-024-00415-y.

DOI:10.1186/s40035-024-00415-y

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11010288/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cb16/11010288/0fd955d5732e/40035_2024_415_Fig1_HTML.jpg

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cb16/11010288/0fd955d5732e/40035_2024_415_Fig1_HTML.jpg

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cb16/11010288/0fd955d5732e/40035_2024_415_Fig1_HTML.jpg

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引用本文的文献

[1]

precision base editing to rescue mouse models of disease.

Mol Ther Nucleic Acids. 2025-7-1

[2]

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本文引用的文献

[1]

Molecularly defined and spatially resolved cell atlas of the whole mouse brain.

Nature. 2023-12

[2]

Tau-targeting antisense oligonucleotide MAPT in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial.

Nat Med. 2023-6

[3]

Tauopathies: new perspectives and challenges.

Mol Neurodegener. 2022-4-7

[4]

Persistent repression of tau in the brain using engineered zinc finger protein transcription factors.

Sci Adv. 2021-3

[5]

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity.

Nat Biotechnol. 2020-3-16

[6]

In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease.

Nat Neurosci. 2019-3-11

[7]

CRISPR/Cas9 editing of APP C-terminus attenuates β-cleavage and promotes α-cleavage.

Nat Commun. 2019-1-3

[8]

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy.

Nat Biotechnol. 2018-4-27

[9]

Distinct differences in prion-like seeding and aggregation between Tau protein variants provide mechanistic insights into tauopathies.

J Biol Chem. 2017-12-19

[10]

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems.

Nat Neurosci. 2017-8

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