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Putting A Patch On Faulty Rna

Developments to Watch


MOST APPROACHES TO GENE therapy involve deleting or replacing faulty genes. But for some genetic disorders, repairs may be possible. That potential is described in the Apr. 28 issue of Proceedings of the National Academy of Sciences by researchers from the University of North Carolina and Bern University in Switzerland.

Messenger RNA molecules ferry information from DNA in a cell's nucleus to its outlying cytoplasm, where proteins are made. Malformed RNA is linked to a number of serious illnesses, including a hemoglobin deficiency called beta-thalassemia. This malady, common in the Middle East and Southeast Asia, can cause anemia and death in children.

Working with modified human cells, researchers were able to correct the problem with molecular bandages. The patches, known as "antisense" molecules, are mirror images of the sites producing faulty RNA. Thus, they fit perfectly over problem sites, blocking the production of defective RNA. In culture, treated cells responded with an increase in globin, a key protein in hemoglobin. The researchers expect to begin animal studies soon.EDITED BY OTIS PORTReturn to top


THE 21ST CENTURY COULD start with a bang for research and development. For the first time in 60 years, civilian R&D could overtake defense technology in Washington's spending priorities. Crossover should occur in fiscal 2000, and by 2003, nondefense research could grab 53% of the federal R&D budget, according to the American Association for the Advancement of Science (AAAS).

Other industrialized countries have long plowed most of their public R&D funds into nondefense work. And competitiveness gurus have been urging the U.S. to follow suit. Adding fuel to the fire, a 1997 study by Chi Research Inc. in Haddon Heights, N.J., turned up strong new evidence that tax-supported research, especially at universities, is crucial to industrial technology.

The AAAS study, which was prompted by the sizable R&D boosts in President Clinton's proposed 1999 budget, does raise some warning flags, though. Most new nondefense funding would go to health and biomedical research. But breakthroughs there often stem from advances in the physical sciences and mathematics--disciplines not slated for big increases. Also, much of the new money would come from tobacco-settlement funds, which are still problematic, politically.EDITED BY OTIS PORTReturn to top

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PHYSICISTS ARE NOW IN HOT PURSUIT OF A "theory of everything" that would merge the theories for quantum mechanics, gravity, and relativity--and unleash a new wave of scientific breakthroughs. The first step is to understand how everything began by recreating the Big Bang of 10 billion years ago, when a tiny "singularity" erupted and gave birth to the subatomic particles that make up matter as we know it.

To simulate the event, physicists at Columbia University have built a special-purpose supercomputer to chew on quantum chromodynamics (QCD) equations--perhaps the toughest problem in quantum physics. Columbia built its first QCD computer in the early 1980s, and Norman H. Christ, head of physics, is sure the new one will cough up the most precise answers yet.

The experiment will get under way by June, running on two home-built supercomputers. Columbia's machine handles 400 gigaflops, or billions of calculations per second. For extra oomph, it will run in tandem with an even faster, 600-gigaflops computer that Christ's crew is still constructing at Brookhaven National Laboratory. Together, they should be able to outstrip earlier QCD simulations, including a 1992 effort by IBM. Big Blue confirmed QCD's predictions in 1993, but only after taking some math shortcuts. By eliminating the shortcuts, Columbia's experiment should be more accurate. Christ figures it will take close to a solid year of number crunching to begin getting answers.EDITED BY OTIS PORTReturn to top

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