Since its discovery several decades ago, gene therapy has been a medicinal sphinx, with doctors as enamored by its
potential as they are frustrated by the riddles it presents. Researchers at the University of Rochester Medical
Center are working to solve one of those riddles.
In order for gene therapy to be effective, doctors must discern how to deliver DNA to a cell's nucleus, which
requires a comprehensive understanding of how DNA and proteins move through cell cytoplasm. Knowledge of this system
could lead to huge leaps in gene therapy effectiveness, and could potentially allow researchers to push forward on
research into many currently-untreatable diseases.
The research is supported by a 4-year, $1.4 million grant from the National Institutes of Health.
One of the best examples is cystic fibrosis, said David A. Dean, Ph.D., professor of Pediatrics and Neonatology at
URMC and the study's lead researcher.
For cystic fibrosis, we know what the affected gene is, and we know what
the mutation is. We know the physiology. The road block is the delivery - getting the corrective DNA in there
and to the right cells for the right amount of time.