Gene therapy replaced missing growth hormone in mice in a preliminary study being presented Monday, June 4, at The Endocrine Society’s 89th Annual Meeting in Toronto.

“The results indicate that gene therapy may be a viable option in the future to treat children who lack growth hormone,” said the study’s lead investigator, Dr. Roberto Salvatori.

Growth hormone deficiency occurs in approximately one in 3,500 children in the United States and slows the growth rate. Without treatment, the child may be very short for his or her age. Currently the only approved treatment involves daily injections of growth hormone. Researchers, therefore, are investigating technologies that may eliminate the need for frequent and potentially painful injections.

Salvatori, an associate professor of medicine at Johns Hopkins University, and his co-authors tested a growth hormone gene by injecting it into young mice that lacked growth hormone. The gene was placed in a non-disease-carrying virus as a carrier. A single injection of the virus caused mice to grow as well as normal mice. Mice that received an inactive substance (placebo) stayed much smaller. Growth hormone was found in virus-treated mice up to six months after injection.

Because the amount of growth hormone lessened with time, Salvatori speculated that the treatment may need to be repeated every six to 12 months. He said that treatments for children are still far off, and that further studies are needed to ensure that gene therapy does not cause liver damage or other complications.

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