使用分子建模开发小型Cas9杂交物种

Antoine Mangin1,2, Vincent Dion3,4, Georgina Menzies5

  • 1UK Dementia Research Institute at Cardiff University, Cardiff, CF24 4HQ, UK.

Scientific reports
|July 26, 2024
PubMed
概括

研究人员探索了较小的Cas9变体和混合体,以感染引起疾病的CAG/CTG重复,用于治疗神经肌肉疾病. 然而,这些工程系统未能实现基因编辑,这表明复杂的Cas9/sgRNA系统目前的in silico设计存在局限性.

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