Monday, October 02, 2006

RNA interference subject of 2006 Nobel Prize in Medicine

Of the citation to Andrew Z. Fire and Craig C. Mello (from AP):

RNA interference opens up exciting possibilities for use in gene technology. Double-stranded RNA molecules have been designed to activate the silencing of specific genes in humans, animals or plants. Such silencing RNA molecules are introduced into the cell and activate the RNA interference machinery to break down mRNA with an identical code.

This method has already become an important research tool in biology and biomedicine. In the future, it is hoped that it will be used in many disciplines including clinical medicine and agriculture. Several recent publications show successful gene silencing in human cells and experimental animals. For instance, a gene causing high blood cholesterol levels was recently shown to be silenced by treating animals with silencing RNA.

(...)
This year's Nobel Laureates have discovered a fundamental mechanism for controlling the flow of genetic information. Our genome operates by sending instructions for the manufacture of proteins from DNA in the nucleus of the cell to the protein synthesizing machinery in the cytoplasm. These instructions are conveyed by messenger RNA (mRNA).


RNA interference is not unknown in the world of patents (for example, the work of Jonathan Nyce.)

Meanwhile, in the world of embryonic stem cell research (from Dr. Jerry Yang (Connecticut) and Dr. Tao Cheng, of the University of Pittsburgh:

Yang's team tried cloning using the blood cells at various levels of development -- from the stem cells stage through full maturity, called full differentiation.

"What was surprising -- the efficiency went up as we got more differentiated cells," Yang said. "That was very, very surprising, very shocking to us."

Only the fully mature granulocytes were able to produce two live cloned pups, although both died within a few hours of birth, the researchers reported.

"Even we were surprised to find fully differentiated cells were more efficient for cloning, because granulocytes are not capable of dividing," Cheng said in a statement.

"In fact, we repeated our experiments six times just to be sure. Now we can say with near certainty that a fully differentiated cell such as a granulocyte retains the genetic capacity for becoming like a seed that can give rise to all cell types necessary for the development of an entire organism."

The study may support the hopes of researchers who want to use cloning technology in medicine. Supporters of so-called therapeutic cloning want to some day be able to take a single cell from a patient, perhaps a skin cell, and use it to generate tailor-made tissue or organ transplants.


On September 30, the Boston Globe wrote:

In 2004, Korean scientist Hwang Woo Suk faked the landmark achievement of extracting the first stem cells from a cloned human embryo. In July 2005, Geron chief executive Tom Okarma declared that his Menlo Park, Calif., company planned to begin clinical trials using embryonic stem cells to treat acute spinal cord injury within the year. Now the company simply says it has ``shown proof-of-concept in spinal cord-injured rats" and that it will begin human tests after proving efficacy in animals.

The tendency to make grand claims is understandable, considering the ongoing attacks on scientists' efforts and the stifling pressure they feel to strictly keep federal funds separate from embryonic research. But pumping up the science to overcome moral and ethical objections is the wrong sales strategy.

Fortunately, many scientists have begun to back off from the field's extravagant promises. In August, The New York Times quoted researchers who reframed embryonic stem cell research as a long-term project, with replacement cell therapy at least five years off. Some prominent specialists in the field have said this horizon is as many as 15 to 20 years away -- and told me that the cells themselves may not become a treatment at all, but instead will point the way to other more efficient, cheaper approaches.

[The Boston Globe also recognized that the ACT work was done in Worcester, MA, not in Alameda, CA:

But despite news of a breakthrough at the company's lab in Worcester, the work didn't live up to the buzz. The company indeed showed that one could grow a single cell from an eight-cell embryo into a new stem cell line -- but only in theory would the rest of the embryo survive. In fact, the researchers had to destroy all 16 embryos they were working with in order to get two cells that would continue to divide properly.]

***
Thomson Scientific had predicted:

Medicine
33% - Chambon, Evans, Jensen
32% - Capecchi, Evans, Smithies
35% - Jefferys

Thus, Thomson Scientific "blew" the Medicine prize and the Physics prize.

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