Early studies have shown how aberrantly expressed microRNAs are a hallmark of several
diseases like cancer. MicroRNA expression profiling was shown to be associated with …

The discovery that gene expression can be controlled by the Watson–Crick base-pairing of
small RNAs with messenger RNAs containing complementary sequence—a process known …

RNA interference (RNAi) is a universal and evolutionarily conserved phenomenon of post-
transcriptional gene silencing by means of sequence-specific mRNA degradation, triggered …

Background: Hereditary cerebellar ataxias (HCA) and hereditary spastic paraplegias (HSP)
are two groups of neurodegenerative disorders that usually present with progressive gait …

The rapid identification of highly specific and potent drug candidates continues to be a
substantial challenge with traditional pharmaceutical approaches. Moreover, many targets …

Since the first description of RNA interference (RNAi) in animals less than a decade ago,
there has been rapid progress towards its use as a therapeutic modality against human …

RNA interference (RNAi) is a powerful approach for reducing expression of endogenously
expressed proteins. It is widely used for biological applications and is being harnessed to …

Improvements in the gene transfer vectors used in therapeutic trials have led to substantial
clinical successes in patients with serious genetic conditions, such as immunodeficiency …

Among the age-dependent protein aggregation disorders, nine neurodegenerative diseases
are caused by expansions of CAG repeats encoding polyglutamine (polyQ) tracts. We …

There are no disease-modifying treatments for adult human neurodegenerative diseases.
Here we test RNA-targeted therapies in two mouse models of spinocerebellar ataxia type 2 …