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April 29, 2024Molecular Therapy — Nucleic AcidsOpen Access

Sequencing-guided design of genetically encoded small RNAs targeting CAG repeats for selective inhibition of mutant huntingtin

Mansi Parasrampuria Adam A White Ramadevi Chilamkurthy Adrian A. Pater Fatima El-Azzouzi Katy N Ovington Philip J Jensik Keith T Gagnon

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Abstract

Huntington's disease (HD) is an incurable neurodegenerative disorder caused by genetic expansion of a CAG repeat sequence in one allele of the

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Cite This Study

Parasrampuria et al. (2024) studied this question.

synapsesocial.com/papers/68e6d064b6db64358764e613 https://doi.org/10.1016/j.omtn.2024.102206

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1BPS2026 – A bacterial assay for screening small molecules that selectively target pathogenic CAG-repeat RNA in Huntington’s diseaseOriginal Research · 2026
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5Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s DiseaseOriginal Research · 2023 · 8 citations