Gene therapy

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Although still a very experimental approach, Gene Therapy techniques are currently being developed to treat and prevent many diseases such as Cystic Fibrosis. This includes many diseases that are as yet incurable, such as cancers and many genetically inherited diseases.

Current approaches being investigated are:

  1. The "knock out" gene approach - Inactivation or removal of genes that have been mutated and/or do not function in the desired way.
  2. The "Replacement" approach - Replacement of disease-causing gene mutations with a healthy copy of the said gene.
  3. The new gene approach - Introduction of a completely new gene into the patient.

Gene Therapy techniques still remain extremely risky and are still very much in the early stages of development, little is known about the side effects of gene manipulation in human patients[1].

There are two forms of gene therapy.

Currently, the form of genetic manipulation research that is supported by the Government using federal funds, this type of gene therapy focuses on repairing malfunctioning genes by inserting new healthy copies of the gene into somatic body cells of an infected individual. Because of this, the therapies are said to act as cures as they eliminate the cause of the disease wholly instead of just relieving the pain from the effects of it. It is, however, not heritable i.e. it cannot be passed on from the patient to his or her future generations.

This form of genetic manipulation acts the same way as somatic therapies, but targets sex cells (egg and sperm cells) instead of somatic body cells. It is seen to be more effective as this type of therapy is heritable and as such, can be passed on from the patient to his or her future generations[2][3].


  1. Genetics Home Reference,What is gene therapy?, published 22/11/10, Last viewed 24/11/10
  2. "Human Health and Diagnostics." Understanding Biotechnology: An Integrated and Cyber-Based Approach. George Acquaah. Prentice Hall, 2003.
  3. "Gene Therapy." Encyclopedia of Science and Religion. Ed. J. Wentzel Vrede van Huyssteen. Vol. 1. Gale Cengage, 2003.
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