An miRNA-mediated therapy for SCA6 blocks IRES-driven translation of the CACNA1A second cistron

Selectively turning off disease genes without disrupting other processes has been a growing goal of genetic research. In a new study, Miyazaki et al . work with a gene that expresses two proteins, a calcium channel, necessary for life, and a regulatory protein, α1ACT, which when mutated causes a form of ataxia called SCA6. These investigators figured out how to block expression of the disease protein without affecting the calcium channel using a small sequence of RNA called miRNA. They then used a viral vector to deliver this miRNA to mice engineered to develop a severe form of SCA6 and successfully prevented the disease.