What is Gene Therapy?


What is gene therapy?

Gene therapy is an experimental technique that uses genes to treat or prevent disease.  OR

The transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders. OR

The treatment of certain disorders, especially those caused by genetic anomalies or deficiencies, by introducing specific engineered genes into a patient’s cells.

These are some of the definitions online. But basically gene therapy simply means the usage of genes as a form of medical treatment instead of using drugs and surgery, for diseases such as cystic fibrosis, combined immunodeficiency syndromes, muscular dystrophy, hemophilia, and many cancers result from the presence of defective genes. Gene therapy can be used to correct or replace the defective genes responsible. It has been especially successful in the treatment of combined immunodeficiency syndromes, showing lasting and remarkable therapeutic benefit

Researchers are testing several approaches to gene therapy, including:

  1. Replacing a mutated gene that causes disease with a healthy copy of the gene.
  2. Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
  3. Introducing a new gene into the body to help fight a disease. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

There are 2 types of gene therapy.

Somatic gene therapy

In somatic gene therapy, the therapeutic genes are transferred into the somatic cells, or the body of a patient. Any modifications and effects will be restricted to the individual patient only, and will not affect the patient’s offspring or later generations. Somatic gene therapy represents the mainstream line of current basic and clinical research.

Germ line gene therapy

In germ line gene therapy, germ cells, i.e., sperm or eggs, are modified by the introduction of functional genes, which are integrated into their genomes. This would allow the therapy to be heritable and passed on to later generations. Although this should, in theory, be highly effective in counteracting genetic disorders and hereditary diseases, many jurisdictions prohibit this for application in human beings, at least for the present, for a variety of technical and ethical reasons.


1.http://ghr.nlm.nih.gov/handbook/therapy/genetherapy                                                                                                                                                             2. http://www.thefreedictionary.com/gene+therapy  3.https://www.google.com.sg/#hl=en&q=gene+therapy&tbs=dfn:1&tbo=u&sa=X&ei=p34LUN3-E4iqrAf6y-zICA&ved=0CG4QkQ4&bav=on.2,or.r_gc.r_pw.r_cp.r_qf.,cf.osb&fp=9601f12d38fee9d4&biw=1366&bih=651                                          4.http://tpx.sagepub.com/content/36/1/97.full


6.http://www.youtube.com/watch?v=gDA6lkoTSeA and Evolution Documentary for uploading the video                                                                                                                                                                                                                                                            .