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Target-agnostic discovery of Rett Syndrome therapeutics by coupling computational network analysis and CRISPR-enabled <i>in vivo</i> disease modeling

2022-03-21

Abstract excerpt

<h4>ABSTRACT</h4> Many neurodevelopmental genetic disorders, such as Rett syndrome, are caused by a single gene mutation but trigger changes in expression and regulation of numerous other genes. This severely impair functions of multiple organs and organ systems beyond the central nervous system (CNS), adding to the challenge of developing broadly effective treatments based on a single drug target. This challenge...

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Literature Corpus work
c67902fc-ae74-541f-ba7c-4a1c5c84a3d6
DOI
10.1101/2022.03.20.485056
Open publication

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Target-agnostic discovery of Rett Syndrome therapeutics by coupling computational network analysis and CRISPR-enabled <i>in vivo</i> disease modelingDOI 10.1101/2022.03.20.485056
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