Gene therapy success

Monday December 12th, 2011

A new gene therapy has successfully been used to treat volunteers with the bleeding disorder haemophilia B, it was announced yesterday.

Volunteers have taken part in marathon running since undergoing the treatment in London, UK, the conference of the American Society of Hematology was told.

Six people took part in the trial at the Royal Free Hospital, London, led by Professor Edward Tuddenham.

The therapy was developed at St Jude Children's Hospital, Memphis, Tennessee, USA, and aims to increase levels of the clotting protein Factor IX.

Following treatment levels of the protein increased from less than one per cent to levels of between two and 12 per cent, the conference in San Diego, California, USA, heard.

Researcher Professor Andrew Davidoff, of St Jude's, said more trials would begin soon.

He said: "The first patient has been followed for the longest time, and his levels have remained at two per cent for more than 18 months.

"These results are highly encouraging and support continued research."

Researcher Dr Amit Nathwani, of University College London, said: "This is a potentially life-changing treatment for patients with this disease and an important milestone for the field of gene therapy.

"It could have ramifications for the treatment of haemophilia A, other protein and liver disorders and chronic diseases such as cystic fibrosis."

Adeno-associated virus vector mediated gene transfer for hemophilia B. New England Journal of Medicine December 10 2011

Tags: Genetics | North America | UK News

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