New Zealand Organisation for Rare Disorders
PO Box 38-538,
Wellington Mail Centre
Phone: +64 4 471 2226
Charities Commission Registration CC22512
Medical Uses of Biotechnology
Some of the things genome knowledge and modern biotechnology make technically possible for human health and disability:
- New tests to confirm a diagnosis of a disease or disability.
This means being able to accurately tell if the person has a particular condition or not.
- New tests to predict a disease or disability.
This means being able to tell if the person has a condition that will lead to the development of certain symptoms. The most well known form of predictive testing is newborn screening, and this is applied to all new babies for a limited range of conditions for which there is some treatment option.
- New tests for susceptibility to a disease or disability.
This means being able to tell if a person has the genetic makeup that means a particular condition is possible or even likely to develop, but in most cases, without any certainty that it will develop, or if it does, how mild or severe it will be.
- New test for carrier status for inherited conditions.
This means being able to tell if the parents or other relatives are carriers of the genetic characteristics that might lead to their children inheriting a particular disease or disability.
- New methods for prenatal testing of a foetus.
This means being able to tell if the foetus that is developing in a pregnant woman, is affected by a particular disease or disability.
- New technology and tests for pre-implantation genetic diagnosis.
This means using tests in conjunction with in-vitro fertilisation techniques, to determine if a fertilised embryo is affected by a particular disease or disability, prior to the implantation of an embryo into the woman so she will become pregnant.
- Enzyme replacement therapy.
This means producing an enzyme that is administered to the person as a medicine, and which takes over the function of a similar enzyme that the person's own cells are unable to produce in the right quantity, as a result of a genetic fault.
- Gene therapy.
This means using genes as medicine, for a person whose own genes have a fault in them. It involves introducing a new gene into the person to compensate for the incorrect expression of their own gene, and may also involve other action to stimulate an inactive gene, or restrict an overactive one.
- Stem cell therapy.
This means using stem cells, which are special cells that have properties that enable them to grow into a variety of different tissue types, and using them to replace or supplement particular tissue in a person with a genetic condition or health problem.
- Xeno-transplantation of cells from animals to humans.
This means taking tissue from other animal species and transplanting it into a person, to replace or supplement particular tissue that is affected by a genetic condition. This could also be used for other purposes such as heart valve replacements or skin grafts.
- Cloning of cells to assist with diagnosis or to improve the effectiveness of other treatments.
This means taking cells and using laboratory techniques to grow more of them to give a good sample to study for diagnosis. Cloning also includes combining part of one cell with part of another cell, to alter the characteristics of the cell that results from this combination.
- Targeted drug therapies.
This means using the precise knowledge of the function of a gene, and the protein or enzyme it produces, to produce a drug that is intended to act only on that particular enzyme or protein, and produce a correction to its absence, deficiency, or excess.