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Getting Healing Genes To The Right Organ

Developments to Watch


A CRITICAL GOAL OF GENE THERAPY is to devise a way to inject a corrective gene into the bloodstream. The gene would then home in on the right organ and go to work, producing a protein to treat or cure a disease. It's a great theory, but getting it to work has been difficult. Now, scientists are nearing their genetic goal in an

inherited disease called ornithine transcarbamylase (OTC) deficiency.

About 1 in 30,000 children are born with the condition, an enzyme deficiency that leaves them unable to convert toxic ammonia into urea, which can lead to coma and death. A team at Philadelphia's Children's Seashore House and the University of Pennsylvania, led by Dr. Mark L. Batshaw, has been working on a method to deliver the gene for the missing enzyme. By periodically injecting the tail veins of mice with a modified virus that carries the gene to the liver, the researchers say they have been able to cure the animals of the disease. Most exciting, they say, the gene therapy continued to work throughout the 10-month lifespan of the mouse, as opposed to just a few weeks. "We've been able to keep the animals metabolically stable for their lifetimes," says Batshaw.

The team has also begun the first human trials of a gene therapy treatment with healthy volunteers, looking for evidence that the added gene is speeding conversion of ammonia to urea. The trials are still in an early stage, but thus far, the treatments show "encouraging results" and no toxic effects, says Batshaw.John CareyReturn to top


MOVIES AND BOOKS SET IN THE FUTURE TEND to depict a world devastated by the pollution generated during the 20th century. But those fictional prognosticators have got it all wrong, say Energy Dept. researchers. They predict that within the next decade we will see environmental gains. Engineered crops will require fewer pesticides and less fertilizer. "Smart" filters will ensure cleaner drinking water. Cars will get 80 miles to the gallon.

Those are among the items identified in the first environmental-technology forecast from Energy's Pacific Northwest National Laboratory in Richland, Wash. The forecast, which Energy hopes to issue annually, was based on a review of technologies in the development or demonstration phase, in both private and public sectors, that should pay off in the next decade. Those deemed most significant were seen as averting problems before they happen, says associate lab director Gerry Stokes.

The No.1 environmental technology, the team decided, will be the genetic engineering of plants. Genetically modified soybeans, tomatoes, and other crops will taste better, require less fertilizer, and resist pests better. Here are the best of the rest, ranked by the researchers in order of importance:

-- Smart water-treatment filters with sensors that will enable them to unclog themselves automatically.

-- Improved storage for electricity from solar and wind power.

-- Microtechnology that will allow everything from chemicals to energy to be used and produced more efficiently. An example: Micro heat pumps will need less energy to warm up rooms.

-- Advanced display systems that will cut down on the use of paper.

-- Molecular design, based on an understanding of how materials behave at the molecular level, that will create efficient solar cells and allow waste-free chemical processing.

-- Bioprocessing that will enable microorganisms to serve as tiny factories for chemicals, fuels, drugs, and enzymes, thereby cutting costs and pollution.

-- Real-time sensors that will identify microbes in air and food immediately.

-- New processes for creating recyclable and biodegradable products.

-- Lightweight, more fuel-efficient cars.EDITED BY CATHERINE ARNSTReturn to top

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