How is siRNA used as therapeutics?
RNA interference (RNAi) is an ancient biological mechanism used to defend against external invasion. It theoretically can silence any disease-related genes in a sequence-specific manner, making small interfering RNA (siRNA) a promising therapeutic modality.
Is siRNA considered gene therapy?
Therefore siRNA can be used to silence genes involved in the pathogenesis of various diseases associated with a known genetic background. As for many neurodegenerative disorders a causative therapy is unavailable, siRNA holds a promising option for the development of novel therapeutic strategies.
Where are microRNAs found?
microRNA is the name of a family of molecules that helps cells control the kinds and amounts of proteins they make. That is, cells use microRNA to help control gene expression. Molecules of microRNA are found in cells and in the bloodstream.
Is siRNA toxic?
The siRNA-induced toxicity described in this work is sequence-dependent, target-independent, reliant on the ability of the duplex to enter an intact RNAi pathway, and in most cases, sensitive to the addition of chemical modifications that minimize off-target effects.
How many siRNA drugs are there?
“Although only three siRNA therapeutics have been approved by the FDA to date, more are sure to follow in the coming years,” the authors wrote. They highlighted the benefits of siRNAs over other novel therapeutic classes.
What is the target of siRNA?
siRNAs that are 21 nt in length or longer, targeting non-mammalian genes, non-expressed genes, non-genomic sequences, pro-angiogenic genes, anti-angiogenic genes and the targets VEGFA and FLT1 all similarly suppressed CNV.
Is antisense therapeutics legit?
Antisense Therapeutics is an Australian publicly-listed biotechnology company, developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases. The products are in-licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS), an established leader in antisense drug development.
How many human miRNAs are there?
There are now over 2000 miRNAs that have been discovered in humans and it is believed that they collectively regulate one third of the genes in the genome. miRNAs have been linked to many human diseases and are being pursued as clinical diagnostics and as therapeutic targets.