miR-19, miR-101 and miR-130 co-regulate ATXN1 levels to potentially modulate SCA1 pathogenesis.
Spinocerebellar ataxia type 1 is caused by expansion of a translated CAG repeat in ataxin1 (ATXN1). The level of the polyglutamine-expanded protein is one of the factors that contributes to disease severity. Here we found that miR-19, miR-101 and miR-130 co-regulate ataxin1 levels and that their inhibition enhanced the cytotoxicity of polyglutamine-expanded ATXN1 in human cells. We provide a new candidate mechanism for modulating the pathogenesis of neurodegenerative diseases sensitive to protein dosage.
Lee
Yoontae
Y
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas 77030, USA.
Samaco
Rodney C
RC
Gatchel
Jennifer R
JR
Thaller
Christina
C
Orr
Harry T
HT
Zoghbi
Huda Y
HY