Why in the News?
Indian researchers have successfully tested a gene therapy for severe haemophilia A, a rare hereditary bleeding disorder.
About Gene Therapy for Haemophilia
- It involves a single infusion of a gene into the liver, which enables it to produce Factor VIII.
- The therapy uses an adenovirus as a vector to deliver the therapeutic gene.
What is Haemophilia?
- A rare, inherited blood disorder that leads to reduced blood clotting, increasing the risk of bleeding and bruising.
- Cause: The body produces insufficient clotting factors, which are proteins that help blood platelets form clots to control bleeding.
- Hemophilia can be severe, moderate, or mild, depending on clotting factor levels in the blood.
- Various forms of it exist, with Haemophilia A (deficiency in Factor VIII) being the most common.
Types of Hemophilia
- Hemophilia A
- Description: Caused by a deficiency of clotting factor VIII.
- Prevalence: The most common type, affecting approximately 10 in 100,000 people globally.
- Key Feature: Often referred to as "classic hemophilia."
- Hemophilia B
- Description: Results from a lack of clotting factor IX.
- Prevalence: Less common, with about 3 in 100,000 people in the U.S. affected.
- Key Feature: Sometimes called "Christmas disease" (named after the first patient identified).
- Hemophilia C
- Description: Caused by a deficiency of clotting factor XI.
- Prevalence: Very rare, affecting only 1 in 100,000 people.
- Key Feature: Unlike types A and B, it can occur equally in both sexes.
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