India conducts clinical trials for ‘haemophilia A’ gene therapy

Blood sample
Haemophilia is usually an inherited bleeding disorder in which the blood doesn’t clot properly. Photo: AFP

• Union Minister Jitendra Singh said that India has conducted the first human clinical trial of gene therapy for ‘haemophilia A’ at Christian Medical College, Vellore.

• Jitendra Singh said 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 that will then express FVIII from specific differentiated blood cells.

• The minister expressed hope that manufacturing of this vector will commence in India soon and proceed with further clinical trials.

Haemophilia A

• Haemophilia 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. 

• The disorder primarily affects males. 

• Haemophilia 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 change or mutation can prevent the clotting protein from working properly or to be missing altogether.

• People with haemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factor in the blood. 

• Hemophilia A is characterised by a deficiency of the blood clotting protein known as Factor VIII that results in abnormal bleeding. 

• Hemophilia B (Christmas Disease) is caused by a lack or decrease of clotting factor IX.

• The frequency and severity of bleeding episodes depends on how much FVIII protein a person produces. Severe hemophilia A is characterised by especially low levels of FVIII (less than 1 per cent in the blood) and represents about 60 per cent of all cases. 

• Severe hemophilia A may result in bleeding into vital body organs such as the kidneys and brain, which can be life-threatening if left untreated.

• Treatment for severe hemophilia A usually involves use of FVIII replacement therapy or an antibody-based medication to improve the ability of blood to clot and reduce the likelihood of bleeding.

The comments posted here/below/in the given space are not on behalf of Onmanorama. The person posting the comment will be in sole ownership of its responsibility. According to the central government's IT rules, obscene or offensive statement made against a person, religion, community or nation is a punishable offense, and legal action would be taken against people who indulge in such activities.