Huntington's disease is caused by an abnormally expanded CAG repeat expansion in the
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …

Small RNAs of 20–30 nucleotides can target both chromatin and transcripts, and thereby
keep both the genome and the transcriptome under extensive surveillance. Recent progress …

Small interfering RNAs (siRNAs) are widely used to study gene function owing to the ease
with which they silence target genes, and there is considerable interest in their potential for …

The recent discovery of microRNAs (miRNAs) took many by surprise because of their
unorthodox features and widespread functions. These tiny,∼ 22-nucleotide, RNAs control …

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

The discovery of miRNAs as important regulatory agents for gene expression has expanded
the therapeutic opportunities for oligonucleotides. In contrast to siRNA, miRNA-targeted …

RNA interference (RNAi) is a natural process through which expression of a targeted gene
can be knocked down with high specificity and selectivity. Using available technology and …

RNA interference (RNAi) was discovered less than a decade ago and already there are
human clinical trials in progress or planned. A major advantage of RNAi versus other …

Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression
and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs …

The transport of RNA molecules from the nucleus to the cytoplasm is fundamental for gene
expression. The different RNA species that are produced in the nucleus are exported …