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Engineering an RNA-based tissue-specific platform for genetic editing through use of a miRNA-enabled Cas12a

2020-03-05

Abstract excerpt

<h4>ABSTRACT</h4> The capacity to edit genomes using CRISPR-Cas systems holds immense potential for countless genetic-based diseases. However, one significant impediment preventing broad therapeutic utilization is in vivo delivery. While genetic editing at a single cell level in vitro can be achieved with high efficiency, the capacity to utilize these same biologic tools in a desired tissue in vivo remains cha...

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Literature Corpus work
227ced50-6072-549b-abb5-41b6757af35a
DOI
10.1101/2020.03.04.977363
Open publication

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Engineering an RNA-based tissue-specific platform for genetic editing through use of a miRNA-enabled Cas12aDOI 10.1101/2020.03.04.977363
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