Recently, the union Science and Technology Minister told that India has conducted the first human clinical trial of gene therapy for ‘haemophilia A’ at Christian Medical College – Vellore.
About Haemophilia A:
It is a genetic disorder that affects the body’s ability to form blood clots.
It is a rare disorder in which the blood doesn't clot in the typical way because it doesn't have enough blood-clotting proteins.
It is a sex linked disorder. The gene for haemophilia is carried on the X chromosome.
Causes: It is 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. This type is caused by a lack or decrease of clotting factor VIII.
Symptoms: Signs and symptoms of haemophilia vary, depending on the level of clotting factors.
Males are much more likely to have haemophilia than are females.
Treatment:
It involves replacement therapy, which involves infusing clotting factor concentrates into the bloodstream to help the blood clot.
Other treatments may include medications to promote clotting or surgery to repair damage caused by bleeding.
What is Gene therapy?
It is a technique that modifies a person’s genes to treat or cure disease.
Gene therapies can work by several mechanisms:
Replacing a disease-causing gene with a healthy copy of the gene
Inactivating a disease-causing gene that is not functioning properly
Introducing a new or modified gene into the body to help treat a disease
Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases.
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