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Trial Therapy May Fight Human Genetic Disorders  — A combination of gene therapy and stem cell therapy may offer a new way to correct inherited diseases, says a University of Florida study.

In research with mice with damaged livers, the scientists removed liver cells called progenitors that have the ability of stem cells to rebuild damaged organs. These progenitor cells were equipped with a healthy human gene and then placed back in the mice. The progenitor cells than began to rebuild the liver with cells that expressed the healthy human gene.


“The idea is that if we use a patient’s own cells, then we don’t have to look for a donor or worry about medications that may be needed to prevent the body from rejecting the therapy,” Bryon Petersen, an associate professor of pathology, immunology, and laboratory medicine, said in a prepared statement.

“We’ve got the ability to basically take cells out of the patient, do our magic and put the corrective gene in, and then put those cells back into the patient. I think it goes back to the concept of self healing self,” Petersen said.

[online issue of Hepatology, 9/04; The Human Genome Project has more about gene therapy. University of Florida, news release, September 2004; 28Sept04 HealthDayNews;ScoutNews]