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Q.5 Explain the concept of gene therapy and its applications in treating
genetic disorders. Describe the use of biotechnology in the development of
new medicines and therapies.
Ans. Gene therapy is a medical technique that involves altering or replacing faulty genes inside
a person’s cells to treat or prevent disease. It aims to fix the root cause of genetic disorders by
introducing healthy copies of genes into a patient's cells, either by directly delivering the gene or
by using modified cells from the patient. This can be done through various methods, such as
inserting a healthy gene into the body’s cells or repairing a malfunctioning gene. One major
application of gene therapy is in treating genetic disorders like cystic fibrosis or muscular
dystrophy, where a single faulty gene causes a disease. For example, in cystic fibrosis, a
defective gene leads to mucus buildup in the lungs. Gene therapy could deliver a healthy gene
to fix the defect, potentially reducing symptoms or preventing the disease from progressing.
Though still in early stages, gene therapy shows promise in offering a long-term solution for
genetic diseases that currently have no cure. In the broader context of biotechnology in
medicine, gene therapy is part of a larger movement to develop more personalized and effective
treatments. Biotechnology also allows for the creation of biopharmaceuticals, such as insulin for
diabetes or monoclonal antibodies for cancer treatment. These treatments are made using living
organisms, such as bacteria or yeast, to produce proteins or other substances that the body
needs to fight disease. Furthermore, biotechnology is revolutionizing vaccines, like the mRNA
vaccines used for COVID-19, which teach the body to recognize and fight viruses without using
live virus material. In cancer treatment, targeted therapies developed through biotechnology are
more precise, focusing on cancer cells while minimizing damage to healthy cells. Overall,
biotechnology is not only helping to develop new medicines but is also paving the way for more
advanced and personalized therapies that could change the future of healthcare.