Ataxia research 4/23
I realize it has been a little quiet on the science and research front, but I hope those of you who attended the NAF conference enjoyed it and saw some progress. I just did a PubMed search on SCA6 and came across this summary of gene suppression studies in SCA mouse models. https://www.mdpi.com/2073-4409/12/7/1037 They chose Dr. Gomez's manuscript to review (...because it is the best... Pastor, P.D.H.; Du, X.; Fazal, S.; Davies, A.N.; Gomez, C.M. Targeting the CACNA1A IRES as a Treatment for Spinocerebellar Ataxia Type 6. Cerebellum 2018 , 17 , 72–77. ) and wrote this: The CACNA1A gene encodes both the α1A protein (pore-forming protein) and the α1ACT protein (transcription factor involved in cerebellar development) [ 39 ]. A polyQ-expanded α1ACT protein is responsible for SCA6, with the α1A protein being essential to life. Therefore, the selective silencing of α1ACT would be the desirable approach, in contrast to full CACNA1A gene silencing [ 39 ]. On the basis that α1AC