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Oh Mickey, You're So Fine For Genetic Research ... (NIH Spends $US8.5 million)
By David King
18mar04

THIS little mouse has four parents and costs $120,000.

It could also hold the key to unlocking some of the world's most debilitating diseases.

The genetically modified rodent is at the cutting edge of a technology being developed in Australia that could identify
genetic causes and remedies to diseases such as cystic fibrosis and spina bifida.

Perth-based research company Ozgene is now making millions of dollars from selling its "knockout" and "knockin" mice to
pharmaceutical companies, academics and research institutes around the world.

Company founder Frank Koentgen explains a "knockout" mouse has a specific gene removed, while a "knockin" mouse has a
mutation made to a specific gene.

The presence or absence of a particular gene can help scientists identify the genetic cause of an illness.

He gives an example: "A family might have an illness like cystic fibrosis, and there is a particular mutation in one of
the genes.

"You make a mouse that has the same genetic mutations; if that mouse gets cystic fibrosis then the gene is one cause of
the condition."

Once identified, the gene becomes a validated drug target, and allows a pharmaceutical company to focus on developing
drugs that act on it. So far, Ozgene mice have been used in research into diabetes, Alzheimer's disease, Parkinson's
disease and auto-immune diseases.

The company recently won a $US8.5 million ($11.5 million) contract to supply its mice to the US National Institute of
Health for the next five years.

The company, in association with the Bone Marrow Research Laboratory in Melbourne, has found one of the genetic causes
of spina bifida.

Dr Koentgen said the mice were one of the most sophisticated tools available in attempts to understand the function of
genes in the human genome.

"These genetically modified mice are generated from embryonic stem cells and a mouse host embryo," he said.

The resulting mouse, which he calls a chimera (an organism composed of two or more genetically distinct tissues), has
four parents: two that produced the stem cell and two that produced the host mouse.

Dr Koentgen, 39, did his doctorate with Roche Pharmaceuticals in Switzerland and, after stints in the US and Canada,
moved to Melbourne to work with eminent immunologist Gustav Nossal at the Walter and Eliza Hall Institute of Medical
Research. He and wife Gabi Seuss founded Ozgene in Perth in 1999.

SOURCE: The Australian, Australia
http://www.theaustralian.news.com.au/printpage/0,5942,8995761,00.html

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