HUMAN GENES PLACED IN STEM CELLS SHOWN TO FUNCTION PROPERLY

MEDIA RELEASE Embargo 10am Tuesday 10 February 2009

HUMAN GENES PLACED IN STEM CELLS
SHOWN TO FUNCTION PROPERLY

Human genes placed in stem cells have been shown to function properly, according to new research at the Murdoch Childrens Research Institute and Melbourne University.

This is the first report showing a full length human gene can be placed in a cord blood stem cell and work properly. This shows stem cells from patients with genetic diseases may be corrected in the lab, giving hope to those living with Cystic Fibrosis and Friedreich Ataxia.

The research found that when a normal copy of a gene was put into a cord blood stem cell, it worked properly when the stem cells form nerve, bone or lung cells in the laboratory. The paper will appear in the next issue of top journal Gene Therapy.

The researchers, led by Dr Faten Zaibak and Professor Bob Williamson, took a full length human gene that codes for a protein known as "frataxin". When this gene does not work properly, affected young people develop Friedreich Ataxia, a debilitating and life-threatening neurodegenerative disorder.

"This gene was introduced into a unique population of cord blood stem cells, which have been shown to form many different types of cells in the body. In some cases, the stem cells took up the gene and switched it on. The protein from the gene was found in the cells just as if it was made from a normal copy of the gene rather than one put there in the lab," Professor Williamson said.

"The stem cells continue to grow and retain the ability to form many cell types in the laboratory, including nerve and lung cells. This is the first report showing a full length human gene can be placed in a cord blood stem cell and work properly, with the stem cell remaining active and able to continue to multiply for months. However, it still remains to be shown that the cells will work if taken from the lab and used for treatment," Dr Zaibak said.

It is universally agreed that using cord blood stem cells is ethically acceptable, and free of the controversy surrounding embryonic stem cells.

"These results suggests that the cord blood stem cells capable of forming many different tissues can be collected from the patient at birth, corrected and then returned to the patient, bypassing any immune complications," Professor Williamson said.




The researchers, who are funded by the Australian Cystic Fibrosis Research Trust and the New Zealand Cystic Fibrosis Association, hope to carry out similar experiments with the cystic fibrosis gene. Currently, the cystic fibrosis gene is difficult to insert into the cell because of its large size.

The authors emphasised that treatment is still in the future, because it is essential to show that the cells can be introduced into a patient in a way that is proven to be safe and effective.