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Gene therapy for haemophilia

18 February 2002
By BioNews
Appeared in BioNews 145

Doctors have successfully used gene therapy in the treatment for a type of haemophilia, a condition caused by a defect in one of the genes which controls blood clotting. Sufferers bruise easily, are prone to nosebleeds, and have a risk of internal bleeding.

A virus that had been genetically altered to carry a working version of the defective gene was transfused into the livers of haemophiliacs. This inserted into the patient's DNA a working version of a gene controlling the manufacture of a blood clotting agent called factor IX. Following the transfusions, the patients were tested to see whether the newly-added gene had caused greater levels of the clotting agent to be produced. The researchers, based at the Children's Hospital of Philadelphia, US, found that levels of factor IX in the blood had risen sharply, enabling them to reduce or stop other treatments the patients were undergoing.

Katherine High, leader of the study, reported the findings at the annual meeting of the American Association for the Advancement of Science in Boston, US. She said that the researchers believe the results are encouraging, adding that 'advances in research and technology suggest that gene therapy will become a successful and powerful method of treating human disease'.


Gene therapy offers cure for haemophilia
The Sunday Times |  17 February 2002
Gene trials lift hope on haemophilia
The Sunday Telegraph |  17 February 2002
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...
14 August 2005 - by BioNews 
US scientists have used gene therapy to successfully treat mice with a rare genetic disorder that causes blindness, called retinoschisis. The researchers, based at the University of Florida Genetics Institute, say their method could eventually be used to treat a range of eye diseases in humans. Retinoschisis causes the retina...
7 June 2004 - by BioNews 
A US biotechnology firm announced last week that it is discontinuing its haemophilia trial, citing technical and safety reasons. Avigen said that it was stopping the trial so it could concentrate on neurological diseases, but it also said that the gene therapy treatment for haemophilia faced 'certain scientific, regulatory and...
6 April 2004 - by Dr Jess Buxton 
Doctors may one day be able to use gene therapy to treat fetuses affected by genetic conditions, say UK scientists. Researchers at Imperial College, University College and the Royal Free Hospital, all in London, have successfully used gene therapy to treat unborn mice affected by haemophilia B. Simon Waddington, of...
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