Discovery of Novel Disease-Causing Mutation in SSBP1 and Its Correction Using Adenine Base Editor to Improve Mitochondrial Function
Molecular Therapy — Nucleic Acids（2024）
关键词
MT: RNA/ DNA editing,mitochondrial diseases,sensorineural deafness,optic atrophy,myopathy,single-stranded binding protein 1,SSBP1,NG-Cas9-based ABE8e,NG-ABE8e,NG-Cas9-based ABE8eWQ,NG-ABE8eWQ,editing efficacy,off-target effects
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