First human clinical trial of gene therapy for hemophilia A (FVIII deficiency)

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.