Gene Therapy

What is Gene therapy?

Gene therapy could be a way to fix a genetic problem at its source. By adding a corrected copy of a defective gene, gene therapy promises to help diseased tissues and organs work properly. This approach is different from traditional drug-based approaches, which may treat symptoms but not the underlying genetic problems.


Most commonly, gene therapy uses a vector, typically a virus, to deliver a gene to the cells where it's needed. Once it's inside, the cell's gene-reading machinery uses the information in the gene to build RNA and protein molecules. The proteins (or RNA) can then carry out their job in the cells.

But gene therapy is not a molecular bandage that will automatically fix any genetic problem. While many disorders or medical conditions can potentially be treated using gene therapy, others are not suitable for this approach.

Pro's and Con's of Gene Therapy

DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information. A gene is a stretch of DNA required to make a functional product such as part or all of a protein. People have about 100,000 to 150,000 genes. During gene therapy, DNA that codes for specific genes is delivered to individual cells in the body.


The Pros:

  • The most important factor in the development of gene therapy is the fact that, for genetic disorders, there is only one way of curing the disease – replacing the defective gene with a healthy copy – and therefore gene therapy is the only hope of finding cures for such disorders
  • If gene therapy targets the reproductive cells of carriers of such genetic disorders as cystic fibrosis, Parkinson’s disease, or cancer, it is possible that any children the carrier goes on to have would be free of the defective gene and on a bigger scale the disease can be wiped out completely
  • Gene therapy, when successful, can have a number of advantages over drug therapy such as providing a cure rather than easing the symptoms. These advantages are discussed in detail in the section ‘Advantages of Gene Therapy’.

The Cons:

Issues based on the science behind gene therapy:

  • The current lack of knowledge and understanding of the treatment means that its safety is unknown. The current scientific understanding is based on theory rather than solid fact. This, however, can be improved with further research and practice.

  • In clinical trials already carried out the effects of the treatment have only been short-lived. To achieve long term results much more research is needed.

  • Drug therapy, although not offering the possibility of a cure, is a tried and tested method and is therefore deemed safer

  • With current knowledge there is no guarantee that the vector carrying the healthy gene will end up in the specific place it is intended – there is a risk of causing even more damage to the genetic make-up that can result in severe consequences for the patient