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Article

RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro.

Nucleic acid therapeutics - 1 Apr 2024

Klementieva Natalia V, Lunev Evgenii A, Shmidt Anna A, Loseva Elizaveta M, Savchenko Irina M, Svetlova Ekaterina A, Galkin Ivan I, Polikarpova Anna V, Usachev Evgeny V, Vassilieva Svetlana G, Marina Valeria I, Dzhenkova Marina A, Romanova Anna D, Agutin Anton V, Timakova Anna A, Reshetov Denis A, Egorova Tatiana V, Bardina Maryana V

Abstract excerpt

RNA interference (RNAi)-based therapeutics hold the potential for dominant genetic disorders, enabling sequence-specific inhibition of pathogenic gene products. We aimed to direct RNAi for the selective suppression of the heterozygous GNAO1 c.607 G > A variant causing GNAO1 encephalopathy. By screening short interfering RNA (siRNA), we showed that GNAO1 c.607G>A is a druggable target for RNAi. The si1488...

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PMID: 38215303
DOI: 10.1089/nat.2023.0043

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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