Better RNA interference, inspired by nature: New nanoparticles offer best-ever gene silencing

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Inspired by tiny particles that carry cholesterol through the body, MIT chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes.This approach, known as RNA interference (RNAi), holds great promise for treating cancer and other diseases. However, delivering enough RNA to treat the diseased tissue, while avoiding side effects in the rest of the body, has proven difficult.The new MIT particles, which encase short strands of RNA within a sphere of fatty molecules and proteins, silence target genes in the liver more efficiently than any previous delivery system, the researchers found in a study of mice.”What we’re excited about is how it only takes a very small amount of RNA to cause gene knockdown in the whole liver. The effect is specific to the liver — we get no effect in other tissues where you don’t want it,” says Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering and a member of MIT’s Koch Institute for Integrative Cancer Research.Anderson is senior author of a paper describing the particles in the Proceedings of the National Academy of Sciences the week of Feb. 10. Robert Langer, the David H. Koch Institute Professor at MIT, is also an author.The research team, which included scientists from Alnylam Pharmaceuticals, also found that the nanoparticles could powerfully silence genes in nonhuman primates. The technology has been licensed to a company for commercial development.Natural inspirationRNA interference is a naturally occurring phenomenon that scientists have been trying to exploit since its discovery in 1998. Snippets of RNA known as short interfering RNA (siRNA) turn off specific genes inside living cells by destroying the messenger RNA molecules that carry DNA’s instructions to the rest of the cell.Scientists hope this approach could offer new treatments for diseases caused by single mutations, such as Huntington’s disease, or cancer, by blocking mutated genes that promote cancerous behavior. However, developing RNAi therapies has proven challenging because it is difficult to deliver large quantities of siRNA to the right location without causing side effects in other tissues or organs.In previous studies, Anderson and Langer showed they could block multiple genes with small doses of siRNA by wrapping the RNA in fatlike molecules called lipidoids. …


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