CSIRO's revolutionary "gene shears" technology has entered clinical trials in adults as the first step in developing a potential treatment for the human immunodeficiency virus (HIV) which causes AIDS, the chief executive of Gene Shears Pty Ltd, Dr Leigh Farrell announced today.
The Phase One clinical trial, conducted at St Vincent's Hospital, Sydney, is testing the safety and ability of Gene Shears' patented gene therapy to inhibit HIV replication in infected patients.
HIV attacks a special class of white blood cell, the CD4+ lymphocyte, which forms the backbone of the human immune defence against attacks by infection. Loss of these white blood cells causes the immune system eventually to break down.
The basis for the trial is the original discovery by CSIRO Plant Industry molecular biologists Dr Jim Haseloff and Dr Wayne Gerlach that naturally-occurring enzymes called ribozymes (dubbed "gene shears") can be used selectively to chop up pieces of genetic material. Gene Shears Pty Ltd went on to prove in laboratory experiments that ribozymesit could be used to prevent the replication of HIV by cutting its genetic material.
The chief executive of CSIRO, Dr Malcolm McIntosh, welcomed news that human trials of the anti-HIV gene shears were under way. "It is extremely pleasing that this fundamental Australian discovery is now starting to bear fruit in the form of practical applications which promise to improve human health and wellbeing," he said.
"It highlights the national importance of CSIRO continuing to perform excellent basic science designed to gain new knowledge."
The Sydney experiment involves six pairs of identical twins, of whom one of each pair is HIV-positive.
CD4+ white blood cells taken from the uninfected twin are equipped with the anti-HIV ribozyme and then infused into the HIV-infected twin.