（1）基因编辑工具的开发与优化：

虽然基于CRISPR的基因编辑技术已经被广泛应用于生命科学研究的各个领域，但它们还有很多缺陷需要进一步改进。例如基因编辑系统一般比较大，这严重影响了在体内的递送；而且现有的基因编辑技术普遍存在潜在的脱靶效应或者编辑效率不够高等问题。在未来的工作中，本研究组将会通过生物信息学挖掘自然界中新型CRISPR基因编辑系统和对现有的基因编辑工具进行改造等方式实现对基因编辑技术进一步优化，希望能够获得更加安全高效的基因编辑工具，为未来的临床应用打下基础。

（2）运用基因编辑技术治疗重大神经系统疾病：

本研究组最终目标是希望将这些新兴的基因编辑技术转化为改善人类健康的方法。神经系统疾病是一类严重威胁人类生活质量和生命的重大疾病，常见的如阿尔兹海默症（AD）或帕金森病（PD）。目前临床上对于很多严重的神经系统疾病都没有根治的方法甚至干预手段也非常有限。我们之前的工作中证明了运用基因编辑技术可以在体内将胶质细胞直接转化为特定类型神经元并缓解了永久性视力损伤和帕金森病的症状。在未来的工作中，我们首先将会应用基因编辑技术在小鼠疾病模型中对重大的神经系统疾病进行治疗，经过小鼠模型的验证之后，我们将会应用到非人灵长类疾病模型。

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