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VIEW: Designer mice —Farrukh Saleem

The leap from a mouse to a man is as big as the Atlantic. The future of medicine, however, belongs to the curing of devastating human illnesses with healthy genes. According to the Nobel Committee, “For the first time in history genetics has become an experimental science”

Three scientists who created ‘designer mice’ have won the 2007 Nobel Prize in Medicine. Mario Capecchi, 70, Martin Evans, 66, and Oliver Smithies, 82, gave us ‘designer mice’ and the Nobel Foundation awarded them the prestigious 10 million Swedish kronor ($1.54 million) Nobel Prize in Medicine.

‘Designer mice’ are genetically engineered mice in which one or more of their genes has been knocked-out in order to study the probable function of that gene. Capecchi, Evans and Smithies first made a particular gene inoperable and then monitored “resulting differences to infer the probable function of that gene (mouse and human genomes are almost identical).”

Bad genetic material is behind all cancers. Heart disease also has genetic origins. Diabetes sufferers also carry a mutated gene. Cystic fibrosis, in which the patient’s “lungs and digestive system face progressive disability” and early death, is also genetic. Muscular dystrophy, a multi-system disorder in which the patient’s heart, nervous system, endocrine glands, skin and eyes face progressive weakness, is also genetic. Alzheimer’s disease, a “degenerative brain disease with progressive memory loss, impaired thinking and disorientation”, has now been traced down to a set of four genes. Parkinson’s disease, a “degenerative disorder of the central nervous system”, is at least partly because of genetic mutation. Arthritis is genetic, so is baldness, and even obesity is said to have a genetic orientation. All in all, bad genetic material is the cause of some 4,000 diseases.

They are showing the world how genes work in mammals and are also leading the way in the removal of bad genes allowing scientists to study how the loss of a gene affects the body and the brain. Designer mice are ushering in “new therapies to correct human genetic defects...therapies that will build on gene modifications in mice that are based on the discoveries of Capecchi, Evans and Smithies.”

Scientists at the University of California (Los Angeles) have now successfully inserted genes into the human brain, a potential for treating Parkinson’s disease. Last year, researchers at the National Institutes of Health in Maryland successfully treated cancer (metastatic melanoma) in two patients by retargeting genes to attack their own cancer cells.

Parker DesLauriers has big, bright blue eyes and chubby cheeks always full of giggles. At six weeks old, Parker was diagnosed with Severe Combined Immunodeficiency (SCID). SCID is a severe genetic disorder in which the patient is extremely vulnerable to every passing virus and infection; average life expectancy at birth of less than a year. Parker could have died from recurrent common cold, chronic diarrhoea or pneumonia. Thanks to ‘designer mice’, Parker is receiving genetically altered stem cells enabling Parker to fight disease. Parker is now two years old.

Currently, 300 to 400 gene-therapy trials are being conducted worldwide. At least half of those trials involve cancer. Experts say that cancer “gene therapies could gain broad use over the next five to ten years” while “genetic therapies for other diseases may be one to two decades into the future.” The University of Pennsylvania School of Medicine has genetically engineered an AIDS virus to fight other AIDS viruses. Baylor College of Medicine in Houston has developed a gene therapy that has successfully cured diabetes in mice.

Admittedly, the leap from a mouse to a man is as big as the Atlantic. The future of medicine, however, belongs to the curing of devastating human illnesses with healthy genes. According to the Nobel Committee, “For the first time in history genetics has become an experimental science.”

Thanks to the creators of ‘designer mice’.

Dr Farrukh Saleem is an Islamabad-based economist and analyst

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