Article

Empirical Simulation and In Silico Evaluation of an Ethically Governed, AI-Enhanced CRISPR Prime Editing Framework for TOR1A Mutation Correction in Dystonia

2025-04-26

Abstract excerpt

Hereditary dystonia, particularly the isolated form of early onset, is often caused by a deletion of the GAG in the TOR1A gene, leading to a dysfunctional TorsinA protein and severe motor impairments. In this study, we investigate an AI-enhanced CRISPR prime editing framework designed for the precise correction of the TOR1A mutation. Our framework integrates the generation of candidate pegRNA with an empirical si...

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Literature Corpus work: 40d02df8-24c5-51b3-be20-623b16e6b271
DOI: 10.1101/2025.04.22.650096

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Empirical Simulation and In Silico Evaluation of an Ethically Governed, AI-Enhanced CRISPR Prime Editing Framework for TOR1A Mutation Correction in DystoniaDOI 10.1101/2025.04.22.650096Read

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