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Jean Erickson, a former preschool director, suffers from Parkinson's disease. Her mobility was improved when doctors drilled a hole into her skull and injected a cold virus modified to help transport a gene into her brain.

The gene created a mini-factory within her cells to pump out dopamine, a chemical that regulates physical movement. "I stopped freezing up," says Erickson, whose treatment was part of research funded by Genzyme (GENZ).

Successes like Erickson's are leading to a revival of interest in gene therapy, which had pretty much dropped off the medical map after the death 11 years ago of an 18-year-old patient. Genzyme and Pfizer (PFE) in the U.S. and Novartis (NVS) in Europe are leading the pack. There are currently 354 U.S. studies under way employing gene manipulation. That's up from 116 worldwide in 2008. San Jose (Calif.) researcher Global Industry Analysts forecasts that sales of gene-based therapies could exceed $465 million annually by the year 2015.

Recent advances in treating brain and eye disorders—and improvements in safety during trials—have led some researchers and investors to compare the current...