Imagine fighting diseases without using toxic medication or risky surgeries. Imagine being able to boost your immune system, or prevent aging, grow stronger muscles, regenerate tissues, and many other wonderful things. All of this might be incubating in the exciting field of biotechnology, where gene therapy is a promising tool for making all sorts of improvements to conventional medical approaches.u

Our body is made out of millions of cells, and cells are made out of millions of biomolecules like proteins, fatty acids, and nucleic acids. The nucleic acids are responsible for the genetic makeup of a particular cell and organism. Nucleic acids contain all the information to build and maintain the cell and DNA is the most famous nucleic acid. In DNA, genes are contained in a somewhat linear sequence. Humans have about 150,000 genes that together comprise all the necessary information for making and maintaining a human being from the embryo stage (of just one cell) to a full-grown healthy adult.

In some cases, a gene may be defective or missing from the DNA sequence of a particular person, causing a deficiency and very often some kind of disease. For example, people who lack the gene to produce melanin (skin pigment) have very pale skin color and are known as albinos. People with hereditary blindness also have one or numerous defective genes related to sight. Sickle cell anemia is another very well-known genetic disorder. The list of genetic disorders is a long one, and in many cases, there is no available cure for the disease.

Gene therapy is defined as the therapeutic delivery of a foreign gene (nucleic acids) to treat a patient’s condition. It is expected that in the near-future medical doctors may treat a wide variety of diseases by inserting genes rather than delivering drugs or practicing surgery.

During the 1980s in a series of studies lead by human genetics scientists of the American national institute of health (NIH) made possible the first successful application of gene therapy in humans. The patient was a 4-year-old girl with adenosine deaminase (ADA) gene deficiency, a condition that made her immune system compromised and unable to fight infections. Doctors corrected the deficiency by placing a DNA containing a normal ADA gene in the white blood cells of the patient. The results were promising, and the child grew to be a normal, healthy human being. Since then many cases of ADA deficiency have been treated in a similar way.

Active research is being done in other genetic conditions and diseases, such as diabetes, cystic fibrosis, cancer, HIV infection, hereditary blindness, and many others.

But gene therapy can also be used to improve a particular function in a healthy human being. For example, increasing muscular mass by introducing a potentiated growth hormone gene. Or reducing/stopping the aging process by delivering genes that regenerate tissues and reverse the natural aging mechanism of the body. All of this may sound like science fiction, but the technology already exists, and most probably in an eyeblink, we will see it permeating into our everyday life.

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