一种针对神经退行性疾病的CRISPR方法。
A CRISPR Approach to Neurodegenerative Diseases.
作者信息
Kampmann Martin
机构信息
Institute for Neurodegenerative Diseases, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA, USA; Chan-Zuckerberg Biohub, San Francisco, CA, USA; California Institute for Quantitative Biomedical Research (QB3), University of California, San Francisco, San Francisco, CA, USA; Quantitative Biosciences Institute (QBI), University of California, San Francisco, San Francisco, CA, USA.
出版信息
Trends Mol Med. 2017 Jun;23(6):483-485. doi: 10.1016/j.molmed.2017.04.003. Epub 2017 May 4.
Abstract
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.
摘要
开发针对神经退行性疾病的有效疗法的一个主要障碍是我们对潜在细胞机制的理解不完整。在人类诱导多能干细胞衍生的神经元中进行基因筛选可以阐明这些机制。使用CRISPR干扰和CRISPR激活的全基因组筛选提供了互补的生物学见解，并可能揭示潜在的治疗靶点。
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