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British doctors have cured youngsters of a deadly inherited disorder using a ground-breaking stem cell treatment which heralds a new dawn for genetic therapies.
Patients with the most severe form of the rare blood condition Wiskott-Aldrich Syndrome have now been free of the disease for four years. Although the disorder affects fewer than one hundred children in Britain, the pioneering therapy offers hope to the hundreds thousands of people suffering from other genetic blood conditions such as sickle cell anaemia.
“We are entering a new era where genetic treatments are entering mainstream medicine and offering hope to patients for whom conventional treatments don’t work well or are simply unavailable,” said Adrian Thrasher, Professor in Paediatric Immunology, at Great Ormond Street Hospital in London.
“The work shows that this method is successful in patients who, in the past would have very little chance of survival without a well match bone marrow donor,” he said.
Read full, original post: New era of medicine begins as first children cured of genetic disorder