A “CRISPR” way to study diseaseJun. 11, 2015, 10:00 AM
by Henry H. Ong
Human diseases often result from complex genetic interactions. A common way to study these interactions is to simultaneously turn off multiple genes, preferably with temporal and spatial precision. However, current methods for this approach, called multiplex conditional mutagenesis, take a large amount of time and resources.
Wenbiao Chen, Ph.D., and colleagues have developed a faster and simpler method. Reporting last month in Genetics, they employed the CRISPR/Cas9 system – a revolutionary technology that greatly accelerates gene manipulation – to perform multiplex conditional mutagenesis in a common human disease model – the zebrafish.
By tailoring the two components of the system, cas9 and guide RNA, to be tissue-specific and inducible, they showed liver-specific mutagenesis of insulin receptor genes in just one generation resulting in impaired glucose homeostasis. They also showed retinal-specific mutagenesis of the ascl1a gene leading to abnormal photoreceptor regeneration in the eye.
This technique can potentially be a valuable tool for advancing our understanding of the genetic basis of human diseases.
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