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What are RNAi Therapeutics?

What are RNAi Therapeutics?

RNAi therapeutics is an innovative new class of medicines based on RNA interference (RNAi)—a breakthrough discovery in understanding how genes are regulated naturally within cells. The significance of the discovery of RNAi was recognized with the 2006 Nobel Prize in Medicine or Physiology.

How siRNA can be used as therapeutics?

The sequence-specific gene-silencing by siRNA can be used as a new therapeutic approach for treatment of a variety of diseases that are incurable by conventional drugs. Many efforts have been made to overcome the problems related to delivery, stability, off-target gene silencing and immunostimulatory effects of siRNA.

What is the function of siRNA?

siRNA is a synthetic RNA duplex designed to specifically target a particular mRNA for degradation. While siRNA provides the opportunity to induce gene knockdown in a variety of cell lines, their utility is limited to cells that are amenable to transfection of synthetic oligonucleotides.

What diseases can be treated with RNAi?

Because of its exquisite specificity and potency, RNAi has attracted a considerable interest as a new class of therapeutic for genetic diseases including amyotrophic lateral sclerosis, Huntington’s disease (HD), Alzheimer’s disease (AD), Parkinson’s disease (PD), spinocerebellar ataxia, dominant muscular dystrophies.

How is RNAi used?

RNAi is widely used by researchers to silence genes in order to learn something about their function. siRNAs can be designed to match any gene, can be manufactured cheaply, and can be readily administered to cells.

When is siRNA used?

Chemically synthesized short interfering RNAs (siRNAs) can be used to induce silencing in cultured mammalian cells. Upon cellular uptake, the guide strand of the siRNA is incorporated into an RNA-induced silencing complex (RISC) that directs degradation of messenger RNAs containing complementary sequences.

Is siRNA a 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.

What is difference between siRNA and miRNA?

Discovered a little over two decades ago, small interfering RNAs (siRNAs) and microRNAs (miRNAs) are noncoding RNAs with important roles in gene regulation. The major difference between siRNAs and miRNAs is that the former are highly specific with only one mRNA target, whereas the latter have multiple targets.

What is the difference between siRNA and RNAi?

RNAi is a sequence specific chain of events. Chemical synthesis of siRNA s is based on coupling of bases to yield a particular sequence.

How does RNAi defend against viruses?

RNAi is a self-defense mechanism of eukaryotic cells, which specially prevent infection evoked by viruses 5. It can inhibit the expression of crucial viral proteins by targeting viral mRNA for degradation through cellular enzymes 9. In fact, RNAi does work effectively as an antiviral agent in plants.

Why is RNAi important?

RNA Interference (RNAi) is one of the most important technological breakthroughs in modern biology, allowing us to directly observe the effects of the loss of function of specific genes in mammalian systems.

How are RNAi therapeutics used to treat disease?

Our RNAi therapeutics mimic this process by delivering specially designed small interfering RNAs (siRNAs) that, as part of RISC (RNA-induced silencing complex), bind to target disease-causing mRNA and guide their destruction like a pair of molecular scissors.

What are the advances in the RNAi platform?

Advances in RNAi Therapeutics Platform 3rd International Conference on the Long and the Short of Non-Coding RNAs Vasant Jadhav, PhD 2 Outline •Introduction to RNAi Platform •New Frontiers for RNAi Therapeutics: CNS and Ocular Delivery •Mechanistic Understanding: Durability of RNAi Therapeutics •RNAi Therapeutics Towards Non-Parenteral Dosing

Which is proof of concept for Alnylam RNAi therapy?

Alnylam Clinical Development Pipeline 1POC, proof of concept –defined as having demonstrated target gene knockdown and/or additional evidence of activity in clinical studies 2Approved in the U.S. for the polyneuropathy of hATTRamyloidosis in adults, and in the EU for the treatment of amyloidosis in adults with stage 1 or stage 2 polyneuropathy

How is RNAi used as an antiviral strategy?

RNAi is a promising antiviral development strategy. Pathways include classic RNAi (mRNA target) and novel RNAi (promoter target). Most RNAi therapeutics act via transient, classical RNAi (cytoplasmic). Chronic human virus infections benefit from heritable, novel RNAi (nuclear). Delivery of RNAi therapeutics is enhanced using nanocarriers.