13 July 2016
Vol 8, Issue 347

About The Cover

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ONLINE COVER microRNAs to the Rescue. The neurodegenerative disease spinocerebellar ataxia type 6 (SCA6) is caused by mutations in the CACNA1A gene, which encodes two different proteins: a calcium channel and the transcription factor α1ACT. Miyazaki and colleagues identify a microRNA called miR-3191-5p that blocks translation of α1ACT (known to drive disease pathogenesis) while sparing calcium channel activity. Taking a gene therapy approach, these investigators show that viral vector delivery of miR-3191-5p into the brains of mice with SCA6 protected cerebellar Purkinje neurons from degeneration (pictured) and prevented mice from developing motor abnormalities such as ataxia, suggesting that this approach may have potential for treating SCA6. [CREDIT: MIYAZAKI ET AL./SCIENCE TRANSLATIONAL MEDICINE]