One of the hardest tasks will be getting RNAi into the body in the first place.
Pseudogene transcripts can be processed into short interfering RNAs that regulate coding genes through the RNAi pathway.
Meanwhile, in the next few months, he expects a "burst of data" that will show RNAi in action.
Merck is using RNAi technology to switch off genes in lab animals it is using in drug discovery.
Maraganore says that much of Alnylam's work will be in figuring out how to get RNAi into the body.
There, the RNAi silenced a gene that was involved in causing their liver cells to die, stopping their disease.
Sirna has laid claim to a huge patent estate in the RNAi field.
RNAi is exciting partly because it shows that some genes work in a way scientists hadn't imagined four years ago.
The first evidence that RNAi can actually treat disease in living creatures was published in today's issue of Nature Medicine.
So the idea behind RNAi therapies is to deliver the chemical signals that throw the switch for production of selective proteins.
Lieberman and her colleagues injected huge amounts of RNAi into the mice, where it was taken up by cells in their livers.
Insert, a subsidiary of Arrowhead Research, is developing the delivery vehicle to carry the RNAi technology of Calandro Pharmaceuticals, another Arrowhead company.
Its role in lymphocyte signaling was characterized and explored through lots of cell-based and in vivo models, including RNAi and other target validation approaches.
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Elledge, a veteran in the RNAi field and display technologies, has come up with a unique way of presenting the human peptidome and screening against it.
And two companies, Alnylam Pharmaceuticals and Sirna Therapeutics, are trying to develop RNAi as a drug against diseases like macular degeneration, cystic fibrosis and pandemic flu.
What's more, later research would reveal that genes that create this kind of RNA--known as RNAi, for RNA interference--existed in plants, fruit flies and even people.
Insert Therapeutics and Intradigm are also working on targeted treatments, but both companies are also exploring the use of a Nobel Prize-winning therapy based on RNA interference (RNAi).
RNAi is another kind of memo entirely--it cancels RNA messages.
But these RNAi bits also hold promise for discovering treatments.
Now a similar sea change may be afoot, thanks to a new technology called RNA interference, or RNAi, which allows scientists to stop a disease by silencing a particular gene.
Using such huge amounts of RNAi could be dangerous in people, and she and her colleagues picked the liver particularly because it would be easy to get the RNAi into the organ.
Insert, a subsidiary of Arrowhead Research (nasdaq: ARWR - news - people ), is developing the delivery vehicle to carry the RNAi technology of Calandro Pharmaceuticals, another Arrowhead company.
Sirna has been in the news of late because Andrew Fire and Craig Mello, the discoverers of the technology upon which Sirna's drugs are based--RNA interference, or RNAi-- won a Nobel Prize for medicine last month.
Thus far, it has 50 issued patents and at least 250 filed patents, many of them gene and viral targets that may be the focus of RNAi drugs developed by Sirna itself or other companies in the future.
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