NIH声明将在未来5年中,以五千两百万美金的资金,赞助一个基因剔除鼠(Knock out Mouse) 的大型合作计划,预期透过这样大型的合作计划,探索完整的基因奥秘,并且将所获得的信息公开,成为未来探究人类疾病的公共资源。
NIH主席Elias A. Zerhouni博士表示,基因剔除鼠是一个非常具有价值的工具,透过特定基因为目标,科学家可以很快的找到基因的功能,加速研究基因与疾病的关系,这样的结果对于疾病的预防以及处理,将在短期内会有惊人的发展。
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相关的研究学者指出,人体细胞里约有两三万个特定的基因,是稳定的在执行基因的功能,而透过基因序列的分析,这些为数众多的基因目标,都可以在老鼠身上找到极为相似的标的,因此利用老鼠特定基因剔除的这个工具,就可以很快的了解特定基因的功能,掌握基因活动的奥秘。
据了解这个起始于NIH的大型研究计划将会与加拿大北美特定基因突变鼠计划(NorCOMM)、欧洲特定基因突变鼠计划 (European Conditional Mouse Mutagenesis Program;简称 EUCOMM)合作,透过这样大型国际性合作的方式,建立起公共基因信息。
英文原文:
Regeneron chosen by NIH for mouse genome project
Regeneron Pharmaceuticals Inc. is one of two groups that will be awarded $47.2 million from the National Institutes of Health to take part in a project that uses mice to determine the role genes play in human disease, the NIH announced today.
Regeneron, based in Eastview, will receive a minimum of $18 million over five years to participate in the NIH Knockout Mouse Project.
As part of the collaboration, researchers will use technologies they developed to remove, or knock out, individual genes from mice to discover what trait each gene expresses. Knocking out each gene in the mouse genome will allow researchers to determine the role each gene plays in human physiology and development and enable scientists to develop better models of hereditary human diseases, such as cancer, heart disease, neurological disorders, diabetes and obesity, the NIH said. The goal is to create a mutation for each of the approximately 20,000 protein-coding genes of the mouse genome, and to make the information publicly available.
"Knockout mice are powerful tools for exploring the function of genes and creating animal models of human disease," NIH Director Elias A. Zerhouni said in a written statement. "By enabling more researchers to study these knockouts, this ... initiative will accelerate our efforts to translate basic research findings into new strategies for improving human health."
So far, researchers have created knockout mice for about 4,000 genes, the NIH said.
Regeneron was selected for its VelociGene technology. Scientists there have been using the process to discover the roles genes play in a wide range of conditions, from obesity to congenital disorders. They are able to create mice with particular diseases and attributes.
Knockout technologies have existed for more than a decade, but Regeneron has found ways to speed up the process and take it further than other competing technologies, said Dr. George Yancopoulos, president of Regeneron Research Laboratories and the company's chief scientific officer.
"It allows us to do things faster and also manipulate much larger pieces of the genome at one time than people could previously manipulate," he said. "We now have the ability to manipulate genes in the mouse at an unprecedented speed and scale."
By replacing large portions of the mouse's immune genes with their human counterparts, for example, Regeneron scientists have been able to create mice with largely human immune systems.
Regeneron's knockout mice exhibit some unusual traits, said Yancopoulos, a Yorktown Heights resident who led in the development of this technology along with David M. Valenzuela, head of the VelociGene division, who is also from Yorktown Heights.
"I have 'mighty mice' that have big muscles that make them look like Arnold Schwarzenegger," he said, referring to mice with extra copies of a gene that regulates muscle growth. "We have thin mice that adjust their metabolism so that the more you feed them ... they rev up their metabolism and stay lean. ... We have red mice because the additional gene is involved in regulating blood vessel development, so they have too many blood vessels."
An early experiment involved adding a gene from florescent algae, which produced a green mouse that glows in the dark.
Yancopoulos said he has shown pictures of these mice to school children to help them understand how genes work.
Other participants in the NIH project will include a consortium comprised of the Children's Hospital Oakland Research Institute in California; the school of Veterinary Medicine at the University of California, Davis; and the Wellcome Trust Sanger Institute in Hinxton, England. In addition, the Jackson Laboratory in Bar Harbor, Maine, will receive $2.5 million for the establishment of a data coordination center.
The participants will work closely with other large-scale efforts to produce knockouts that are under way in Canada and Europe.
"The international projects will exchange information and coordinate their efforts in much the same way that teams from many nations collaborated on the International Human Genome Project," said Colin Fletcher, a program director at the National Human Genome Research Institute, in a statement. The institute, part of the NIH, will oversee the administration for much of the Knockout Mouse Project.
"This is the inheritance of the human genome sequencing project — this is the next step," Yancopoulos said.
Regeneron is a small biopharmaceutical company that has several drugs in clinical trials for the potential treatment of cancer, eye disease and inflammatory disease. Yancopoulos said this grant should help the company even further.
"This will help put us at the forefront of the companies that are working to take advantage of the human genome sequence — to first figure out gene function and then to take advantage of this information to develop new drugs that address important, unmet medical needs," he said.