How does rnai work

A simplified model for the RNAi pathway A simplified model for the RNAi pathway is based on two steps, each involving ribonuclease enzyme. The high degrees of efficiency and specificity are the main advantages of RNAi. You are here: NCBI. External link.

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Manager, Reagent Manufacturing. Gene expression is the process by which a set of instructions in DNA are converted into proteins. They build and repair tissues; are critical to the development of the brain, heart, lung, liver, bones, muscles, cartilage, blood, and skin; and make up many body chemicals including enzymes and hormones. The genetic code for making proteins contained in the DNA directs the synthesis of a version of RNA called messenger RNA, or mRNA, which transports these instructions out of the nucleus of the cell where DNA resides into the cytoplasm of the cell where it is used by ribosomes to make new proteins.

In other instances, our approach delivers therapeutic benefit by using siRNA to target and improve the levels of other, non—disease-causing proteins that are implicated in the disease pathway ie, those that are not caused by a single genetic mutation. This second approach allows for the RNAi therapeutics to be used in a broader base of human diseases, such as hypercholesterolemia and hypertension. Additionally, we believe that siRNAs can be developed to address infectious diseases by directly targeting viral RNA or their host factors for destruction such that the virus is unable make copies of itself or to get inside cells in the first place.

Solving for delivery was one of the primary obstacles that Alnylam scientists had to overcome in pioneering this new class of medicines.