Biotechnology is a field of biology which plays an important role in the life of various organisms and species. Gene therapy is a biotechnological experimental technique in which the gene of any organism is operated, in order to treat and prevent many diseases. It is also useful for the replacement of an abnormal gene with a healthy one and the production of some beneficial protein.
Nowadays, Genetic diseases are treated genetically by the means of inserting a required gene in the cells of the affected person or organisms instead of using any surgery or drugs. The biotechnological advancement had made many things possible. The insertion of the required gene works wonder. Scientists are searching and testing several approaches to gene therapy which includes:
- The replacement of the muted gene with a healthy one
- Eradication or inactivation of the mutated gene
- Insertion of a strong gene which helps to fight a particular disease
There is two main and successful example of gene therapy.
- a) Somatic gene therapy:
The first illness to be treated by the gene therapy is called “Severe Combined Immuno Deficiency Disease (S.C.I.D)”, characterized by the poor immune system so the person may suffer from severe infections such as pneumonia, influenza, etc, and consequently die. Patients suffering from such condition cannot synthesize an important enzyme, which is called Adenosine-Deaminase (ADA). Such patients can be treated by removing defective cells from the bone marrow and inserting the normal gene for ADA enzyme into those cells and then inserting back these treated cells into the patients’ bone marrow. As a result of this, genetically altered cells start to produce their own ADA and provide normal immune to the patient. As Somatic cell gene therapy only targets the affected gene, therefore, the alteration in the gene are not passed on to the offspring.
- b) Germ line gene therapy:
This therapy deals with the modification of the DNA in a patient’s germ cells (eggs or sperm). Germ-line therapy is also very helpful for the treatment of the cystic fibrosis. In this disease, patient produces excessive mucus due to the defect in the gene. In this condition, a liposome (microscopic lipid envelope) would be sprayed periodically with a healthy gene on the lung epithelia of the patient. Liposome packages will be taken up into the cells of the lungs’ surface then the helpful genes may enter the chromosome and express themselves.
Gene therapy is a very useful treatment for many diseases, including hereditary disorders, cancers, cell mutation, and certain viral infections. However, this treatment is quite risky; researchers are keen to study this treatment more and to make it more effective and safe. Science students are mostly assigned to write science
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