StemCell Therapy

This type of therapy is called therapeutic gene therapy or the use of genes as medicine. It is an experimental form of treatment that is still being developed, but it has the potential to revolutionise treatment for all kinds of genetic conditions.

Gene therapy targets the faulty genes responsible for genetic diseases. Inheriting a faulty (mutated) gene can directly cause a wide range of disorders such as cystic fibrosis and haemophilia. It can also cause susceptibility to some cancers. Gene therapy can be used to replace a faulty gene with a healthy version or to introduce a new gene that can cure a condition or modify its effects. This type of gene therapy is called therapeutic gene therapy or the use of genes as medicine. It is an experimental form of treatment that is still in its infancy but has the potential to revolutionise treatment for all kinds of genetic diseases.

Inheriting one or both copies of a faulty gene can cause a wide range of conditions such as haemophilia and cystic fibrosis, and can also result in increased susceptibility to some cancers. Gene therapy targets the faulty genes responsible for a genetic condition. Gene therapy can be used to replace a faulty gene copy with a working version or to introduce a new gene that can cure a condition or modify its effects.

One promising technique is to put the working gene inside a harmless virus, which has had most of its own genes removed it has been deactivated. A virus that causes disease (such as the common cold) works by slipping into a cell, taking over its DNA and forcing it to produce more viruses. Similarly, a deactivated virus can enter the specific cell and deliver the working gene.

Other techniques involve using stem cells. These are immature cells that have the potential to develop into cells with different functions. In this technique, stem cells are manipulated in the laboratory to accept new genes that can then change their behaviour. For example, a gene might be inserted into a stem cell that could make it better able to survive chemotherapy. This would be of assistance to those patients who could benefit from further chemotherapy following stem cell transplantation.

To make sure that future generations of the persons family were not affected by the genetic condition, their germ cells would need to undergo gene therapy too. However, a complicated range of ethical issues, as well as technical problems, means that gene therapy of germ cells is only a remote possibility.

The majority of trials are being conducted in the US and Europe, with only a modest number initiated in other countries, including Australia (1.6%). Most trials focus on treating acquired conditions such as cancer and AIDS, although an increasing number of genetic conditions are being targeted.

The concern is that manipulating factors such as intelligence might be tried, once gene therapy becomes commonplace. Ordinary characteristics, such as shortness or average IQ, might then be considered subnormal.

Another concern is that gene therapy might only be available to the rich. The challenge for nations experimenting with gene therapy is to come up with workable, fair and ethical guidelines for its use.

This page has been produced in consultation with and approved by: Better Health Channel - (need new cp)

Last updated: May 2011

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Gene therapy - Better Health Channel

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