A major barrier to developing effective therapies for neurodegenerative diseases is our incomplete understanding of the underlying cellular mechanisms. Genetic screens in human-induced pluripotent stem cell-derived neurons can elucidate such mechanisms. Genome-wide screens using CRISPR interference and CRISPR activation provide complementary biological insights and may reveal potential therapeutic targets.

中文翻译：

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针对神经退行性疾病的CRISPR方法

开发针对神经退行性疾病的有效疗法的主要障碍是我们对基本细胞机制的不完全了解。人类诱导的多能干细胞来源的神经元的遗传筛选可以阐明这种机制。使用CRISPR干扰和CRISPR激活的全基因组筛选可提供互补的生物学见解，并可能揭示潜在的治疗靶点。