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A new hope in haemophilia through gene therapy

New Delhi, Tue, 13 Dec 2011 NI Wire

Haemophilia is considered an incurable disease and caused by a genetic flaw in which the patient's (suffering from Haemophilia B) blood does not clot and continuous flows even a small cut due to absence of factor IX (FIX) in the blood. The patients survive lives at potentially high risk, and can be dead if not find the medical emergency on time.

There was no permanent cure as it happens due to missing a gene in patients' genetical structure. However, a new study conducted by a team of University College led by an Indian origin researcher has shown a ray of hope in treatment of the Haemophilia B.

According to Amit Nathwani, lead researcher of the team, 'they have succeed to install working genes in human body that cure the problem of FIX and patients? blood began to clot.'

'However, this is not cure but it can pave the way of new researches in this field,' said Dr. Ronald G. Crystal, another gene therapist at Weill Cornell Medical College.

This research was conducted over mere six patients who have below 1% of normal levels of FIX in their blood and it was published on New England Journal of Medicine on 10 December 2011.

Generally genetical engineering is being used to treat some rare genetical disorders. Some researches have also been conducted to cure Haemophilia in which a working gene was introduced in the body using cold virus. The concept behind inserting the working gene in the body is that it will fulfill the missing gene and cold virus does not effect to the body, but every times the efforts failed because our immune system attack the inserted foreign bodies and killed the virus before beginning the working genes to function.

But, Nathwani and his team had successfully installed the working genes in the blood using genetic engineering in more efficient way. The result was exciting as after following six to sixteen months, most of the patients? FIX levels rose to 3% to 11% of normal values.

Out of six patients, two patients each received low, intermediate or high doses of the gene therapy in an IV-like infusion in the arm.

(With Agencies Input)


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