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Alnylam Pharmaceuticals
Alnylam Pharmaceuticals is a biopharmaceutical company headquartered in Cambridge, Massachusetts. The company’s core focus is the development and commercialization of novel therapeutics based on RNA interference, or RNAi, for genetically defined diseases. Alnylam was founded in 2002 by Phillip Sharp, Paul Schimmel, Dave Bartel, Thomas Tuschl and Phillip Zamore.
==RNAi Explained==
(詳細はRNAi is a natural mechanism for silencing specific genes. Genes provide cells with the instructions for making proteins, and proteins — or more specifically proteins made abnormally — are the cause of many human diseases. When a gene is silenced, the cell stops making the protein specified by that gene, thereby improving the course of the disease.
RNAi was first discovered in worms in 1998.〔("Potent and specific genetic interference by double stranded RNA in Caenorhabditis elegans". ) FIRE, A.; XU, S.; MONTGOMERY, M.K.; KOSTAS, S.A.; DRIVER, S.E. and MELLO, C.C. , Nature, 1998, vol. 391, no. 6669, p. 806-811.〕
In 2001, Alnylam founders began using small interfering RNAs, known as siRNAs, to silence genes in mammalian cells.〔("Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells" ) ELBASHIR, S.M., HARBORTH, J., LENDECKEL, W., YALCIN, A., WEBER, K. & TUSCHL, T. (2001). Nature 2001, vol 411, pp: 494-8.〕 Human proof of concept has been demonstrated by Alnylam, including in a Phase I trial which resulted in statistically significant reduction of a protein called transthyretin, or TTR. Alnylam has demonstrated human efficacy with intravenous and subcutaneous modes of administration.
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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