Trinucleotide Repeat Sequestration via CRISPR-Guided RNA Targeting

Trinucleotide Repeat Sequestration via CRISPR-Guided RNA Targeting proposes using RNA-targeting CRISPR systems (CasRx/Cas13d or dPspCas13b) to selectively bind and neutralize toxic expanded repeat RNA transcripts without degrading them — a "sequestration" approach that prevents the pathological RNA gain-of-function mechanisms driving Huntington's disease, myotonic dystrophy, and fragile X-associated tremor/ataxia syndrome while preserving some residual protein production from the targeted transc...