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Gene therapy progress

13 December 1999
By BioNews
Appeared in BioNews 38

Genetic therapy is becoming a realistic treatment for a number of disorders. At the meeting of the American Society for Haematology in New Orleans, advances in gene therapy for two disorders were reported. These were X-linked severe combined immunodeficiency (SCID) and haemophilia B. In SCID, affected individuals have virtually no T lymphocytes or natural killer (NK) cells and the only treatment is bone-marrow transplantation. Marina Cavazzana-Calvo (Hopital Necker, Paris) reported that in the two patients with the longest follow-up, T lymphocytes and NK cells were increased to normal numbers and normal T-lymphocyte function achieved. Although the results are preliminary, this may represent the first cure of a genetic disease by gene therapy. Researchers also reported in last week's issue of Circulation the successful use of gene therapy to revive failing heart cells. So far, the therapy has worked only in hearts that have been removed from the patients. The researchers report that getting the gene to take in a living body still needs more work. The team, which includes Dr Sian Harding of Imperial College London, based the trials on the gene SERCA2a which instructs cells to produce a protein that is involved in controlling heart muscle contraction. In this particular trial, the cells from the failing hearts began beating and contracting at almost normal levels.

Gene gets failing hearts beating again
The Daily Telegraph |  7 December 1999
Gene-therapy advances greeted positively
The Lancet |  11 December 1999
Successful gene therapy on hemophilia and heart cells reported |  7 December 1999
21 September 2009 - by Dr Will Fletcher 
The key gene that causes blood stem cells to become the immune system's disease-fighting 'Natural Killer' (NK) cells has been identified for the first time. UK researchers were studying the gene responsible, named E4bp4, to investigate its effects on a rare but fatal form of childhood leukaemia when they stumbled upon its apparent role as a master switch for the production of NK cells. The scientists, from University College London, Imperial College London, and the...
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