朝向一个通过 CRISPR 理解人类大脑发育中基因功能的目标。

Toward a CRISPR understanding of gene function in human brain development.

机构信息

Institute for Neurodegenerative Diseases, University of California, San Francisco, San Francisco, CA, USA; Biomedical Sciences Graduate Program, University of California, San Francisco, San Francisco, CA, USA.

Institute for Neurodegenerative Diseases, University of California, San Francisco, San Francisco, CA, USA; Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA, USA.

出版信息

Cell Stem Cell. 2023 Dec 7;30(12):1561-1562. doi: 10.1016/j.stem.2023.11.005.

DOI:10.1016/j.stem.2023.11.005

PMID:38065064

Abstract

Genome-wide association studies pinpoint genetic risk factors for neurodevelopmental disorders (NDDs), but the next challenge is to understand the mechanisms through which these genes affect brain development. Two recent CRISPR screens in human brain organoids interrogate the function of risk genes for autism spectrum disorder and other NDDs.

摘要

全基因组关联研究确定了神经发育障碍 (NDD) 的遗传风险因素，但下一个挑战是了解这些基因影响大脑发育的机制。最近两项在人类大脑类器官中的 CRISPR 筛选研究探讨了自闭症谱系障碍和其他 NDD 的风险基因的功能。