By John Denny
Staff Reporter
(St Kitts) – A local research facility has made a scientific breakthrough in gene therapy that could possibly lead to cures for a number of diseases.
The study, conducted by the St. Kitts Biomedical Research Foundation, showed for the first time in primates, that the function of a gene can be safely silenced.
Genes are the master blueprints for the body and largely control development as well as many bodily functions throughout life. Since the discovery of many defective genes related to hereditary diseases and cancers, efforts have been made to replace them or to silence them using “gene therapy.” None of the efforts to silence genes has been effective and safe in a primate, until now.
“As far as we are aware this is the first time this has been done,” said Dr. Milton Whittaker of the Foundation. “Of course this is only a first step. It will be quite some time before the full potential of this can be put to use.”
The study was published March 27 in the world’s leading scientific journal, Nature. The new study showed that injection of a gene-silencing compound, termed a “locked nucleic acid,” could turn off a gene that controls cholesterol production in the liver. Remarkably, administration of just three doses over the course of one week markedly altered cholesterol levels for seven weeks, resulting in a significant decrease in total cholesterol, with no evidence of toxicity.
“All medicines currently used for cholesterol have some level of toxicity associated with them,” Dr. Whittaker said.
The research team was led by Matthew S. Lawrence, M.D., Ph.D., at St. Kitts Biomedical, and included Sakari Kauppinen from the University of Copenhagen, other scientists from Denmark and the United States, and many members of the research and animal care staff at St. Kitts Biomedical.
“The demonstration that these compounds are stable and active in African green monkeys opens many possibilities for targeting other gene sequences to explore biological pathways and to treat diseases,” said Dr. Lawrence.
The facility uses local Green Vervet monkeys in its research. Special traps are used to ensure they are not injured when being caught.
The St. Kitts Foundation’s investigators were the first to prove that new genes could be inserted into the brain of a primate in 1998 and have been studying gene therapy for Parkinson’s disease under two United States Public Health Service grants since that time under the leadership of foundation president Dr. Eugene Redmond.
This newly- published study is the “proof of principle” that the findings from the human “Genome Project” can be translated into actual treatments for diseases, many of which are untreatable at the present time, Dr. Redmond said.
“Many additional studies will be needed in monkeys to develop these treatments using this exciting technology,” Dr. Redmond noted.
The St. Kitts Biomedical Research Foundation has been operating in St. Kitts since 1982 and has become one of the world’s leading biomedical research centers using non-human primates. Its research activities are supported by numerous grants from the United States Public Health Service, by the Michael J. Fox Foundation for Parkinson’s Research, Project ALS, RxGen, Inc., and the Axion Research Foundation of Connecticut.
Affiliated scientists come from more than a dozen universities in the United States and Europe.
