Patisiran
Patisiran (pronounced puh-TIS-ih-ran) is a medication used in the treatment of hereditary transthyretin-mediated amyloidosis (hATTR). It is marketed under the brand name Onpattro by Alnylam Pharmaceuticals.
Etymology
The name "Patisiran" is derived from the drug's mechanism of action. It is a siRNA (small interfering RNA) that is used to silence the expression of a specific gene, in this case, the gene that produces transthyretin (TTR). The "pati-" prefix is a reference to the drug's "pathway" of action, and "-siran" is a common suffix for siRNA drugs.
Usage
Patisiran is used to treat the polyneuropathy caused by hATTR, a rare, inherited disease that results in the buildup of abnormal amyloid protein in the body's organs and tissues. It is administered via intravenous infusion.
Mechanism of Action
Patisiran works by silencing the TTR gene, reducing the production of the TTR protein that forms amyloid deposits in hATTR patients. This is achieved through a process called RNA interference (RNAi), where the siRNA molecule binds to the TTR mRNA, preventing it from being translated into protein.
Related Terms
See Also
External links
- Medical encyclopedia article on Patisiran
- Wikipedia's article - Patisiran
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