Context:
Recently, India conducted 1st human clinical trials for ‘haemophilia A’ gene therapy.
Hemophilia A:
- A rare genetic disorder
- It affects the body’s ability to form blood clots
- The blood doesn’t clot in the typical way because it doesn’t have enough blood-clotting proteins
- A sex-linked disorder – The gene for hemophilia is carried on the X chromosome
- Males are much more likely to have hemophilia than are females
- Caused by – A mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot
- Symptoms – Signs and symptoms of hemophilia vary, depending on the level of clotting factors
- Treatment –
- Replacement therapy – Involves infusing clotting factor concentrates into the bloodstream to help the blood clot
- Medications to promote clotting
- Surgery to repair damage caused by bleeding
Gene therapy:
- A technique that modifies genetic material of a person to treat or cure diseases or medical disorder
- It works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patient’s cells with a healthy version of that gene
- Diseases treated with gene therapy –
- Inherited genetic diseases – Hemophilia and sickle cell disease
- Acquired disorders – Leukemia
- Several mechanisms in Gene Therapy –
- Replacement of disease-causing gene with a healthy copy of the gene
- Inactivation of disease-causing gene that is not functioning properly
- Introduction of new or modified gene into the body to help treat a disease
- Used studied to treat diseases including – Cancer, genetic diseases, and infectious diseases
Read more about: Autoimmune diseases
Source: Indian Express
Previous Year Question
Consider the following statements:
1. Genetic changes can be introduced in the cells that produce eggs or sperms of a prospective parent.
2. A person’s genome can be edited before birth at the early embryonic stage.
3. Human induced pluripotent stem cells can be injected into the embryo of a pig.
Which of the statements given above is/are correct?
UPSC Civil Services Exam – 2020 Prelims]
(a) 1 only
(b) 2 and 3 only
(c) 2 only
(d) 1, 2 and 3
Answer: (d)
