Article

Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins

2022-07-24

Abstract excerpt

<h4>ABSTRACT</h4> Genome editing of somatic cells via clustered regularly interspaced short palindromic repeats (CRISPR) offers promise for new therapeutics to treat a variety of genetic disorders, including neurological diseases. However, the dense and complex parenchyma of the brain and the post-mitotic state of neurons make efficient genome editing challenging. In vivo delivery systems for CRISPR-Cas proteins...

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Literature Corpus work: d27468a3-c7d8-5a21-a215-c1dba9619914
DOI: 10.1101/2022.07.24.501299

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