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Primate Models Used in Gene Research

October 3, 2003

Heather Heilman
Phone: (504) 865-5714

hheilman@tulane.edu

"The gene therapy effort at Tulane is a good example of what happens when you hire good people and give them what they need to be successful," said Bruce Bunnell. "It's been highly successful in a short time."

bunnellBunnell is head of the new gene therapy division at the Tulane National Primate Research Center, which is the first primate center in the nation to have a division devoted to gene therapy. It's still a pretty small operation, consisting of Bunnell and a staff of five in a new building at the primate center. But it's already made significant progress.

Bunnell's academic appointment is in the pharmacology department, so he is in the unique position of having three bosses--Darwin Prockop at the Center for Gene Therapy, Andrew Lackner at the primate center, and Krishna Agrawal in the pharmacology department.

But there's so much excitement about gene therapy at Tulane that everyone is eager to cooperate. Bunnell was attracted to Tulane by the opportunity to work with Darwin Prockop on the stem cell approach to gene therapy. He had worked with gene therapy at the National Institutes of Health and Ohio State University before coming to Tulane.

That approach uses a virus to deliver genes to a patient's cells in order to correct those genes that cause disease. His lab is currently focused on two lyosomal storage diseases, Tay-Sachs and Krabbe's, a disease for which the primate center has developed the only primate model.

The advantage with working with primates is that the results should be directly translatable to humans. This has not always been so of trials conducted in mice. Bunnell is using both the traditional viral vector and the stem cell approach to target these diseases.

"My thinking is there will be a need to take stem cells from a patient who has a genetic disease, genetically correcting those cells, and putting them back in the body to become lung cells or brain cells," he said. "We need both technologies, but stem cells are where the excitement is right now in research," he added. "The explosion in the literature is amazing, even in the short time I've been at Tulane. If you'd done a literature search a year ago, you might have gotten a thousand papers. Now it's up to several thousand papers."

One of his division's significant contributions to the field has been to isolate stem cells from three sources in the rhesus monkey--cells from the bone marrow, brain and fat tissue. "Now we can look at the efficiencies of these three different cell populations, and because they originated from the same animal they act as a control for themselves," he said.

In other words, researchers should be able to tell if stem cells that came from the brain are better able to become brain cells. Until now, scientists have not been certain that differences that they see aren't attributable to variations between animals.

"Now we need to study the biology and find out what these cells are capable of," Bunnell said. "I have ambitions of applying them to the Krabbe's model. It's a difficult disease to correct because it affects both the central nervous system and the peripheral nervous systems, and the traditional vector-based approach has not had much success."

He hopes to make the cells they've isolated available to researchers at other institutions. "If someone wants these cells, we want to let them have access to them, because if they can prove they function, it moves the field forward."

Heather Heilman can be reached at hheilman@tulane.edu.

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