TY  - JOUR
AU  - Feng, Weijun
AU  - Liu, Hai-Kun
AU  - Kawauchi, Daisuke
TI  - CRISPR-engineered genome editing for the next generation neurological disease modeling.
JO  - Progress in neuro-psychopharmacology & biological psychiatry
VL  - 81
SN  - 0278-5846
CY  - Amsterdam [u.a.]
PB  - Elsevier Science
M1  - DKFZ-2018-00026
SP  - 459 - 467
PY  - 2018
N1  - DKFZ-ZMBH-Allianz
AB  - 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.
LB  - PUB:(DE-HGF)16
C6  - pmid:28536069
DO  - DOI:10.1016/j.pnpbp.2017.05.019
UR  - https://inrepo02.dkfz.de/record/131720
ER  -