DNA discoveries

Gene sequencing could reveal clues to diseases, produce new drugs

By Carly Shulaka and
Christine L. Romero


Life will never be the same.

††† The unraveling of the human genome, the total of all the genes in our 43 chromosomes is bound to transform medicine.

†† For businesses planning to commercialize information flowing from the Human Genome Project, itís bound to be lucrative, if theyíre willing to make the long-term high-stakes investment. The project is an international effort to sequence the entire DNA of human cells by 2005. Analyzing the estimated 100,000 human genes could bring an end to diseases, such as AIDS and cancer, and halt the aging process.

†††† "Itís going to change diagnostics and therapeutics," said Ed Brody, vice president of drug discovery at Boulder, Colo., based NeXstar Pharmaceuticals. "Itís going to change everything about how we do medicine."

†††† The federally funded Human Genome projectís price tag is about $3 billion, but the price is dropping as technology improves. In comparison, the federal government will spend more than $200 billion on defense this year. The Human Genome Project is headed by the National Institutes of Health and Department of Energy. Recently, some private companies have joined the race and say they will map the genome faster and cheaper than the government.

†† The potential for U.S. commercial development spawning from the genome project is estimated at more than $20 billion by 2000, according to Oak Ridge National Laboratory, a technology laboratory by Lockheed Martin for the Department of Energy.

†† "The Human Genome Project is a work in progress," said Ralph Chistofferson, president and chief executive officer of Ribozyme Pharmaceuticals (RPI).

†† "The good news is the sequences are going much faster. The bad news is that 90 percent or more of the partial gene sequences have no known function. That doesnít mean they donít have a function. It means we havenít figured out what they are yet."

†† RPI is using the research to develop new drugs. "It takes five to 10 years to develop a drug," he said. "It is high cost - it is common to hear people say it costs at least $100 million for each new product.

†† "Your chances of success are probably about 10 percent, but the payoff could be huge."

†† Humans and chimpanzees are nearly identical genetically. Scientists believe that less than two percent of our DNA is different from that of chimps. One difference is a genetic component that makes chimpanzees immune to AIDS and humans susceptible to the progress of the HIV-virus.

†† Genoplex Inc. researchers are comparing the genetic makeup of chimps and humans to find the genetic code in humans responsible for the susceptibility to AIDS.

†† Eventually the company plans to partner with a pharmaceutical company to develop new drugs based on the findings.

†† Other biotech firms are developing ways to analyze the Genome Project data. NeXstar is working to develop a machine that can come up with potential targets for the genes as they are being sequenced, according to Ed Brody, NeXstarís vice president for drug discovery.

†† "We think the Human Genome Project will not be the big breakthrough," he said. "The big breakthrough will be when we know what proteins code for which genes and when we are able to detect in blood the levels of proteins. In blood, once we know all of the proteins, we will see things in the blood one cannot even imagine."

†† At Genomica, Thomas G. Marr, president and CEO, said he is developing software to help analyze the Human Genomeís projectís data. The software helps scientists find genes in two ways; either using diseased genes or using model organisms. The most common model organisms used in genetic research are mice, yeast, worms, fruit flies and E. coli. Despite the obvious differences between the organism and humans, the genomes are similar and are a cost-effective way to study the inheritance of genes through many generations over a short period of time.

†† "I think itís important if not to provide the data in the public domain to provide it at a very reasonable level of access to the public domain," Marr said. "Everybody needs a chance to look at it and interpret it and try to figure out what the sequence means.

†† "As the sequencing of the human genome progresses, there will be 3 billion (genomic bases) out there, and people need to try and figure out what they do. Itís a monumental task."

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