First human clinical trial of gene therapy for hemophilia A (FVIII deficiency)
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First human clinical trial of gene therapy for hemophilia A (FVIII deficiency)
Subject: Science and tech
Section: Health
Context:
- India has conducted the first human clinical trial of gene therapy for haemophilia A (FVIII deficiency) at Christian Medical College (CMC) Vellore.
More on news:
- The trials involved deploying a novel technology of using a lentiviral vector to express a FVIII transgene in the patient’s own haematopoietic stem cell which will then express FVIII from specific differentiated blood cells.
About Haemophilia:
- Hemophilia is usually an inherited bleeding disorder in which the blood doesn’t clot properly.
- This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.
- Blood contains many proteins called clotting factors that can help to stop bleeding.
Types of Haemophilia:
Congenital hemophilia
- Hemophilia is usually inherited, meaning a person is born with the disorder (congenital). Congenital hemophilia is classified by the type of clotting factor that’s low.
- The most common type is hemophilia A, associated with a low level of factor 8 The next most common type is hemophilia B, associated with a low level of factor 9.
Acquired hemophilia
- Some people develop hemophilia with no family history of the disorder.
- This is called acquired hemophilia.
- Acquired hemophilia is a variety of the condition that occurs when a person’s immune system attacks clotting factor 8 or 9 in the blood.
Hemophilia inheritance
- In the most common types of hemophilia, the faulty gene is located on the X chromosome. Everyone has two sex chromosomes, one from each parent.
- Females inherit an X chromosome from the mother and an X chromosome from the father. Males inherit an X chromosome from the mother and a Y chromosome from the father.
- This means that hemophilia almost always occurs in boys and is passed from mother to son through one of the mother’s genes.