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All-in-one AAV-delivered epigenome-editing platform: <i>proof-of-concept</i> and therapeutic implications for neurodegenerative disorders

2023-04-16

Abstract excerpt

Safely and efficiently controlling gene expression is a long-standing goal of biomedical research, and the recently discovered bacterial CRISPR/Cas system can be harnessed to create powerful tools for epigenetic editing. Current state-of-the-art systems consist of a deactivated-Cas9 nuclease (dCas9) fused to one of several epigenetic effector motifs/domains, along with a guide RNA (gRNA) which defines the genomic...

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Literature Corpus work
dfcf75aa-155e-5836-ae63-ca4cca0b9378
DOI
10.1101/2023.04.14.536951
Open publication

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