Haemophilia A

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.