Adelaide Scientists Make HIV A Safe Aid In Gene Therapy

Many human diseases result from missing or damaged genes in our cells. Such abnormal or missing genes can potentially be replaced by ‘normal’ genes using the technique of ‘Gene Therapy’, thus overcoming the disease.

Senior researcher Dr Don Anson and PhD student Maria Fuller from the Women’s and Children’s Hospital, Adelaide have today published a paper in Human Gene Therapy in which they have shown that Human Immunodeficiency Virus Type 1 (HIV-1), after modification can be safely used to transfer therapeutic genes into human cells without transferring the disease.

“Viruses are very good at transferring their genetic material into cells, so it is logical to use them to carry any therapeutic genes we want to get into those same cells. HIV-1 is particular suited to this purpose, because, firstly, the genetic material transferred becomes a permanent part of the host cell, and secondly because it transfers genetic material to cells that are not growing and not all viruses can do this – and it is these non growing cells that we need to target with therapeutic genes,” Dr Anson says.

“However to use HIV-1 for this purpose we must be sure it cannot infect our cells and cause AIDS. We are now able to modify HIV’s genetic material so those viral proteins essential for it to reproduce and cause disease in our cells are removed.”

Other groups worldwide have also been working on gene transfer using HIV-1, but Dr Anson’s group has developed a method to vastly increase the safety with which HIV-1 can be used to transfer genes without causing disease.

Currently animal models of genetic disease are being studied using this approach before the methods can be taken to trials of human disease.

The use of this HIV-1 transfer approach has wide applicability for a variety of human genetic diseases.

This work was supported by the National Health and Medical Research Council.