CRISPR-based epigenetic editing of Gad1 improves synaptic inhibition and cognitive behavior in a Tauopathy mouse model.

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Tác giả: Henry Y Feng, Pei Li, Yong Ren, Lei Wan, Wei Wang, Zhen Yan, Mingjun Yu, Ping Zhong

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Neurobiology of disease , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 497314

GABAergic signaling in the brain plays a key role in regulating synaptic transmission, neuronal excitability, and cognitive processes. Large-scale sequencing has revealed the diminished expression of GABA-related genes in Alzheimer's disease (AD), however, it is largely unclear about the epigenetic mechanisms that dysregulate the transcription of these genes in AD. We confirmed that GABA synthesizing enzymes, GAD1 and GAD2, were significantly downregulated in prefrontal cortex (PFC) of AD human postmortem tissues. A tauopathy mouse model also had the significantly reduced expression of GABA-related genes, as well as the diminished GABAergic synaptic transmission in PFC pyramidal neurons. To elevate endogenous Gad1 levels, we used the CRISPR/Cas9-based epigenome editing technology to recruit histone acetyltransferase p300 to Gad1. Cells transfected with a fusion protein consisting of the nuclease-null dCas9 protein and the catalytic core of p300 (dCas9
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