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Gene therapy hope for haemophiliacs

11 June 2001
By BioNews
Appeared in BioNews 111

A preliminary safety study has shown that gene therapy may be able to help sufferers of haemophilia. The study, results of which were published in the New England Journal of Medicine last week, involved six people with severe haemophilia A, a disorder caused by the failure of a gene called factor VIII which codes for a protein necessary for blood clotting.

The researchers, from the Beth Israel Deaconess Medical Centre in Boston, USA, modified cells taken from skin on the patients' upper arms by adding the Factor VIII gene taken from healthy humans, and grew them in a laboratory. Between 100 million and 400 million cells were then reimplanted into each of the patients.

After four months, four of the patients had higher levels of the gene in their blood than before the treatment, but not enough for them to forgo their usual treatment which involves intravenous infusions of the protein. No factor VIII was detected in any of the patients after ten months. The researchers do not know whether the implanted cells died or just ceased to function.

The study undertaken used only a small number of patients, but Dr David Roth of the Boston Centre is encouraged by the results. He said that the findings suggest that 'gene therapy has a major potential to transform the way we care for patients with haemophilia'. Other scientists remain unconvinced both by the temporary nature of the results and the disparity among the outcomes in the patients. Dr Inder Verma from the Salk Institute in San Diego noted that two of the patients did not respond to the treatment and that implanting more cells did not alter this.

Cell transplants help haemophiliacs
The Washington Post |  7 June 2001
Gene therapy helpful for haemophiliacs - at least in theory
LA Times |  11 June 2001
Genetic work eases haemophilia
BBC News Online |  6 June 2001
Study finds nonviral gene therapy for sever hemophilia a is safe with early signs of benefit
EurekaAlert |  6 June 2001
11 July 2011 - by Dr Caroline Hirst 
Scientists have, for the first time, successfully treated a blood disorder by repairing errors in the DNA of a living animal. Researchers from The Children’s Hospital of Philadelphia, together with California-based Sangamo BioSciences, have applied an innovative genome editing technique to treat haemophilia B, which affects around one in 30,000 boys and men...
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