%0 Journal Article
%A Feng, Weijun
%A Liu, Hai-Kun
%A Kawauchi, Daisuke
%T CRISPR-engineered genome editing for the next generation neurological disease modeling.
%J Progress in neuro-psychopharmacology & biological psychiatry
%V 81
%@ 0278-5846
%C Amsterdam [u.a.]
%I Elsevier Science
%M DKFZ-2018-00026
%P 459 - 467
%D 2018
%Z DKFZ-ZMBH-Allianz
%X Neurological disorders often occur because of failure of proper brain development and/or appropriate maintenance of neuronal circuits. In order to understand roles of causative factors (e.g. genes, cell types) in disease development, generation of solid animal models has been one of straight-forward approaches. Recent next generation sequencing studies on human patient-derived clinical samples have identified various types of recurrent mutations in individual neurological diseases. While these discoveries have prompted us to evaluate impact of mutated genes on these neurological diseases, a feasible but flexible genome editing tool had remained to be developed. An advance of genome editing technology using the clustered regularly interspaced short palindromic repeats (CRISPR) with the CRISPR-associated protein (Cas) offers us a tremendous potential to create a variety of mutations in the cell, leading to 'next generation' disease models carrying disease-associated mutations. We will here review recent progress of CRISPR-based brain disease modeling studies and discuss future requirement to tackle current difficulties in usage of these technologies.
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:28536069
%R 10.1016/j.pnpbp.2017.05.019
%U https://inrepo02.dkfz.de/record/131720