Research Direction

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Gene editing techniques & Gene therapy for genetic brain disorders

CRISPR-Cas systems have revolutionized gene editing researches and represent promising techniques for gene therapy. However, CRISPR-based tools still face great challenges including restricted activity windows, off-target effects andin vivodelivery efficiencies. Here we focus on the development of novel gene editing tools and improvement of available CRISPR-based tools to expand their applications. We also focus on their potential applications as gene therapy tools for genetic diseases and will assess the therapeutic effects using mouse models of neurodevelopmental disorders.

CRISPR-Cas systems have revolutionized gene editing researches and represent promising techniques for gene therapy. However, CRISPR-based tools still face great challenges including restricted activity windows, off-target effects andin vivodelivery efficiencies. Here we focus on the development of novel gene editing tools and improvement of available CRISPR-based tools to expand their applications. We also focus on their potential applications as gene therapy tools for genetic diseases and will assess the therapeutic effects using mouse models of neurodevelopmental disorders.

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