CRISPR/Cas9 facilitates investigation of neural circuit disease using human iPSCs: mechanism of epilepsy caused by an SCN1A loss-of-function mutation
Mutations in SCN1A, the gene encoding the α subunit of Nav1.1 channel, can cause epilepsies with wide ranges of clinical phenotypes, which are associated with the contrasting effects of channel loss-of-function or gain-of-function. In this project, CRISPR/Cas9- and TALEN-mediated genome-editing tech...
Main Authors: | , , , , , , , , , , , , , , , , , , |
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Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2016
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5068877/ |