Gene Editing for Kidney Disease
February 26, 2019 | Terry Sharrer
Joubert syndrome is a rare genetic neurological disorder that is more frequent in Ashkenazi, French Canadian and Hutterite populations. The causative mutation inhibits primary cilia that sense physical environments and chemical signaling—consequently producing many strange symptoms. To treat Joubert in vitro (human kidney cells) and in mouse models, researchers at Newcastle University used a gene editing technique called “exon skipping” to “rescue” the truncated protein from deformed cilia. MORE
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