COLLEGE PARK, MARYLAND, April 29, 2009 -- In a recent issue of The Journal of Chemical Physics, published by the American Institute of Physics (AIP), a group of researchers at the University of California, Berkeley and Los Alamos National Laboratory describe the first comprehensive, molecular-level numerical study of gene therapy. Their work should help scientists design new experimental gene therapies and possibly solve some of the problems associated with this promising technique.
"There are several barriers to gene delivery," says Nikolaos Voulgarakis of Berkeley, the lead author on the paper. "The genetic material must be protected during transit to a cell, it must pass into a cell, it must survive the cell's defense mechanisms, and it must enter into the cell's guarded nucleus."
If all of these barriers can be overcome, gene therapy would be a valuable technique with profound clinical implications. It has the potential to correct a number of human diseases that result from specific genes in a person's DNA makeup not functioning properly -- or at all. Gene therapy would provide a mechanism to replace these specific genes, swapping out the bad for the good. If doctors could safely do this, they could treat or even cure diseases like cystic fibrosis, certain types of cancer, sickle cell anemia, and a number of rare genetic disorders.
Safety is a primary concern when working with gene therapy. Some of the first attempts at gene therapy used viruses to insert DNA into cells -- something that viruses naturally do anyway. Viruses can be dangerously toxic, however, and this fact was tragically demonstrated a decade ago when an 18-year-old boy enrolled in a gene therapy study had a massive immune reaction to the viruses used. He died just a few days into the treatment from multiple organ failure, precipitating an immediate halt to the trial.
Since then, many alternatives to viruses have emerged for use in gene therapy
|Contact: Jason Bardi|
American Institute of Physics