Therapeutic strategy of neurodegenerative disease using microRNA

我们在培养细胞实验中发现了对神经病理学脊髓小脑变性和亨廷顿病（SCD） mRNA稳定性影响最大的rna结合蛋白。在细胞培养实验中，我发现CUGBP是一种rna结合蛋白，Elav-like family （CELF）抑制SCD基因和HD发病机制的mRNA表达。此外，这种表达抑制作用并不独立于SCD和HD基因特异性发病机制的阵列长度（CAG重复数）。目前，我们制备了SCD和HD小鼠模型，并阐明了CELF蛋白影响SCD和HD发病基因mRNA表达的机制，即表达CELF蛋白的AAV，检测其治疗效果。

We identified RNA-binding proteins that influence most strongly on the stability of the mRNA of Neuropathology spinocerebellar degeneration and (HD) Huntington's disease (SCD) in cultured cell experiments. In cell culture experiments, I found that CUGBP is an RNA-binding protein, Elav-like family (CELF) suppresses the mRNA expression of pathogenesis of SCD gene and HD. In addition, this expression suppression effect was not independent of the length of the array length of the gene-specific pathogenesis of SCD and HD in (CAG repeat number). Currently, it is administered to a mouse model of SCD and HD prepared and elucidation of the mechanism by which CELF proteins affect the expression of mRNA of pathogenesis gene of SCD and HD, the AAV expressing the CELF proteins, we examined the therapeutic effect.