Optimizing twin prime editing components for scalable genome editing and therapy in spinocerebellar ataxia type 3

Abstract excerpt

Recent advances in prime editing technologies using CRISPR modules fused with reverse transcriptase (RT) have enabled efficient and precise reprogramming of target genomic sequences. Twin prime editing using two coordinated prime editor complexes is a promising strategy for inducing extensive genomic modifications via reverse transcribed complementary templates. However, current twin prime editing systems still re...

Topics

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• CRISPR and Genetic Engineering
• CRISPR-Cas Systems
• Gene Editing
• Genetic Therapy
• Mutation
• Phenotype
• Point Mutation
• Virus-based gene therapy research

Identifiers and source

Literature Corpus work

568b62e0-e6c2-5329-8c75-e80c970e3bae

DOI

10.1101/2025.07.22.666117