Haemophilia A

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Source-This post on Haemophilia A is based on the article “India conducted 1st human clinical trials for ‘haemophilia A’ gene therapy: Science and Technology minister” published in “The Indian Express” on 28th February 2024.

Why in the News?

Recently, India has conducted the first human clinical trial of gene therapy for ‘haemophilia A’ at Christian Medical College – Vellore.

What is Haemophilia A?

Haemophilia A
Source- Medicineplus

1. About– It is a genetic bleeding disorder caused by a deficiency in clotting factor VIII (blood-clotting protein), which is necessary for blood clotting.

2. Sex-linked:
a.
It is a sex-linked disorder determined by the X and Y chromosomes. The gene for hemophilia is found on the X chromosome.
b. Hemophilia is much more common in males than in females.

3. Symptoms– Prolonged bleeding after injuries, surgeries, or spontaneous bleeding into muscles and joints.

4. Treatment: 
a. It includes replacement therapy, where clotting factor concentrates are infused into the bloodstream to assist blood clotting.
b. Other treatments may involve medications to encourage clotting or surgery to mend bleeding-related damage.

Most common types of Hemophilia :
1. Hemophilia A (Classic Hemophilia)– This type is caused by a lack or decrease of clotting factor VIII.
2. Hemophilia B (Christmas Disease)– This type is caused by a lack or decrease of clotting factor IX.

What is a Gene therapy?

1. Gene therapy is the introduction, removal or change in genetic material (typically means DNA and RNA) in the cells of a patient to treat an inherited or developed disease.

2. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve human body’s ability to fight disease.

Read moreGene therapy

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