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Gene therapy presents potential treatments to disease

Universtiy of Rochester Medical Center

Medical researchers are putting a lot of resources into understanding genes as both the causes and solution to many diseases.

University of Rochester Medical Center professor of pediatrics Dr. David Dean studies the best way to treat diseases through gene therapy.

His laboratory recently received a  four-year, $1.4 million grant from the National Institutes of Health.

Dean explains one challenge facing researchers in his field.

"The main roadblock we've had to almost all the gene therapy techniques has been this one of delivery," Dean said. "How do you get it into cells and ultimately into humans?"

Dean’s research seeks to find more efficient delivery systems for gene therapy, with potential implications for many diseases that are currently untreatable.

"What we find true in the cells that we're using holds true in the lung, as well as it holds true in the brain if we're doing delivery," Dean said. "So, on the one hand, it applies to everything."

Dean’s laboratory also focuses much of its attention on lung diseases, including acute lung injury which causes fluid buildup in the lung. It can cause death between 25 and 30 percent of the time.