发信人: raison (), 信区: Biology
标 题: 多转些好文章吧
发信站: Unknown Space - 未名空间 (Thu Jan 20 22:15:58 2005) WWW-POST

个人认为，不能傻干，讨论讨论好的IDEA，要上好JOURNAL，没有NOVELTY，光JBC，EMBO
恐怕不行啊
‘
我带个头，此文是GENE
DELIVERY/THERAPY领域，IDEA是用CHOLESTEOL做载体把一分子的CHOLESTEOL附加在RNAi送
入造脂细胞（特异性摄取CHOLESTEROL）达到高效降脂作用。
我研究NEURODEGENERATIVE DISORDER，正在琢磨啥东东能被APOPTOTIC NEURON特异性摄取
，把RNAi attach 到该东东上就可以达到NEUROPROTECTION的效果。请大家畅所欲言吧


RNA treatment lowers cholesterol

sofner 投递 "News@Nature-Nov 10,2004-RNA treatment lowers cholesterol

Cholesterol levels have been lowered in mice by using tiny pieces of genetic
material to block a particular gene. The demonstration shows that the
experimental technique, called RNA interference (or RNAi), could be a
practical way of curing a wide range of diseases.

Scientists have had a hard time finding a convenient way to deliver the
treatment to patients so that it reaches the right part of the
body.Researchers from the biotechnology company Alnylam, based in Kulmbach,
Germany, and Cambridge, Massachusetts, modified the RNA molecule used to
trigger the interference by attaching a cholesterol molecule to it.

RNAi works by using small molecules of RNA to trigger a cell to shut off a
particular gene, for example, one that codes for a harmful protein. Clinical
trials that use RNAi to treat an eye disease called macular degeneration have
already begun.

But scientists have had a hard time finding a convenient way to deliver the
treatment to patients so that it reaches the right part of the body. In the
macular degeneration trials, for example, scientists inject the treatment
directly into the eye.

Researchers from the biotechnology company Alnylam, based in Kulmbach,
Germany, and Cambridge, Massachusetts, have figured out a way to solve the
problem, at least for treating high cholesterol. They modified the RNA
molecule used to trigger the interference by attaching a cholesterol molecule
to it.

Cells that make cholesterol have cholesterol receptors on their surface, so
that they can sense levels of the molecule in the blood. Adding the
cholesterol molecule to the RNA ensures that the complex is taken up
specifically by these cells, in the liver and small intestine.

When the researchers injected this modified molecule into mice, they found
that it cut the animals' levels of 'bad' cholesterol by 44%. They report their
results in this week's Nature1.

Simple solution

Another concern about RNAi has been whether the therapy might affect genes
other than the ones targeted, causing side-effects. The researchers checked
the activity of a few genes unrelated to their target gene, and found that
they were not affected.

The company is excited about the result. "We view our work as a historic step
forward in the development of RNAi therapeutics as a potential new class of
drugs," says John Maraganore, head of Alnylam.

He says the company aims to develop similar methods to treat other sorts of
diseases, such as diabetes, obesity and cancer. "There are clearly many
diseases of interest here," he says.

Other researchers agree that the new method has removed a major roadblock that
could have prevented RNAi from moving to the clinic.

"This is a fairly simple solution to a problem that could have been immense in
this field," says John Rossi, a molecular biologist at the Beckman Research
Institute of the City of Hope in Duarte, California.

But he points out that Alnylam still needs to overcome many practical issues
before its research in mice can be translated into patients. The doses used in
the mice were quite high, and would be costly and impractical to give to
people. And it's not clear how often a person would need to be injected with
the treatment, or whether it could be delivered in some other form.

Alnylam says it is working on these issues, and hopes to design more effective
molecules that could be used in smaller doses in the future. In the meantime,
it will file a clinical trial plan with the Food and Drug Administration next
year, which will use RNAi to treat an eye disease. If its plan is approved,
Alnylam will be the third company so far to ask the federal agency to approve
an RNAi therapy.

News
http://www.nature.com/news/2004/041108/full/041108-11.html
References
http://www.nature.com/nature/journal/v432/n7014/pdf/nature03121.pdf"





--
※ 来源:．Unknown Space - 未名空间 mitbbs.com．[FROM: 67.71.]