BioengineeringMolecular Therapy — Nucleic AcidsPeer-reviewed
Optimizing twin prime editing components for scalable genome editing and therapy in spinocerebellar ataxia type 3
Lee Wha Gwon, Jung Bae Seong, Hyeon-Gu Yeo…·Seung Hwan Lee
This study optimizes twin prime editing components to improve genome editing efficiency and accuracy for therapeutic applications in spinocerebellar ataxia type 3.
DOI 10.1016/j.omtn.2026.102988