Therapy for Genetic Disorders

Most genetic disorders, unfortunately, cannot be cured. The available treatments help manage the diseases caused by abnormal genes, while the treatment itself and its efficacy vary from one type of disorder to another. Genetic researchers, however, are very optimistic about gene therapy which has shown promising results in clinical trials. However, it remains unavailable to the wider population and at the moment of writing, gene therapy is used only for clinical trials.
Gene therapy is hoped to cure or improve treatment of genetic disorders by replacing the mutated or malfunctioned gene, manipulating or turning off the gene causing the disease or stimulate other bodily functions to fight the disease. The most common method is replacement of a malfunctioned or sometimes a missed gene with a healthy one. However, gene therapy poses a risk of potentially serious complications, in the first place due to the method that is used to insert the “new“ genes – the use of viruses. These have the ability to identify certain cells as well as to transmit the genetic material into the cells containing malfunctioned or missed gene. For that reason modified viruses are used as vectors or carriers of the healthy genes. This method of insertion of healthy genes may not seem problematic at a first glance but it can cause cause potentially serious complications as already mentioned earlier.

  • Drug treatments
  • Enzyme replacement therapy
  • Gene therapy
  • RNAi therapies

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