What is it about?

This study demonstrates that a novel CRISPR/Cas9 gene-editing delivery system can bypass the blood–brain barrier and regulate neuronal receptor pathways to treat chronic anxiety. Five weeks after intranasal delivery of the vector and package, 75 mice were tested with standard behavioral assays measuring mouse anxiety. Mice treated with the CRISPR package showed a significant reduction in anxiety compared with control mice. Our results suggest that noninvasive, intranasal delivery of CRISPR/Cas9 therapeutics may help patients with anxiety.

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Why is it important?

39 million Americans suffer from anxiety disorders and ~30-40% do not respond to current treatments including the SSRIs, Zoloft and Cipramil. What if I told you Cognigenics has developed a new therapeutic, RCA-101, that can be given as a single dose intranasally, and permanently lower your anxiety? Our recently accepted article in the top-tier journal, PNAS-Nexus, has demonstrated proof-of-principle of this therapeutic by harnessing the power of CRISPR to modulate a serotonergic system in the brain.

Perspectives

For me personally, having anxiety and trying to find new treatments for anxiety has been a personal journey that has been very rewarding as a result of my connection both in research and my own anxiety.

Troy Rohn

Read the Original

This page is a summary of: Genetic modulation of the HTR2A gene reduces anxiety-related behavior in mice, PNAS Nexus, May 2023, Oxford University Press (OUP),
DOI: 10.1093/pnasnexus/pgad170.
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