Gene Therapy Fixes 'Incurable' Disorder of the Immune System
Steve Connor Science Editor, The Independent (London, England)
Gene therapy has successfully repaired the immune systems of two men who were born with an "incurable" genetic disorder that rendered them almost defenceless against bacterial and fungal infections.
Sixteen months after an operation to repair a defective gene, which each man had inherited from his mother, the once severely ill patients show signs of complete recovery.
The latest success with gene therapy will lead to further operations to treat many other chronic and incurable conditions resulting from the inheritance of defective genes, scientists believe.
Gene therapy involves the alteration of a person's genome to repair or replace a mutated gene. After many years of hype and hope, the technique seems finally to have proven its potential with a handful of successful operations.
The latest success was achieved by doctors in Germany, Switzerland and Britain who treated two men with chronic granulomatous disorder (CGD), an immunodeficiency disease caused by a defective gene on the maternally inherited X chromosome.
The men - both in their mid-twenties - suffered repeated life- threatening infections from bacteria and fungi and from an early age had to rely on powerful antibiotics and antifungal medicines. Since their operations 16 months ago at the University Hospital in Frankfurt, both patients have gained weight and have been free of the severe infections that have plagued them since childhood.
Manual Grez of the Institute for Biomedical Research in Frankfurt, one of the leaders of the study published in the journal Nature Medicine, said the results suggest that it may be possible to use gene therapy to treat other similar inherited diseases.
"This a landmark study for chronic granulomatous disorder and the potential of gene therapy. These are very positive results but there is still a lot of work …
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Publication information: Article title: Gene Therapy Fixes 'Incurable' Disorder of the Immune System. Contributors: Steve Connor Science Editor - Author. Newspaper title: The Independent (London, England). Publication date: April 3, 2006. Page number: 6. © 2009 The Independent - London. Provided by ProQuest LLC. All Rights Reserved.
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