A New Era Of Medicine: Gene Therapy Cures Rare Inherited Diseases

British doctors have cured a rare genetic disorder for some patients using stem cells - a result that could usher in a new era of medicine.
Danny, Sarah and Michael Wheeler at home: Patients with the most severe form of a rare blood disease called Wiskott-Aldrich Syndrome are now past 4 years without any symptoms.
Although less than 100 children in the UK have the disorder, it offers hope for thousands of patients with other genetic blood diseases such as sickle cell anemia.
Daniel Wheeler, 15, is among seven children who are being tested for gene therapy at several London facilities and Paris.Daniel was diagnosed at 2 years old and had to constantly be hospitalized because of symptoms including Severe eczema, asthma and loss of resistance to infection

. One of my brothers died from complications of the disease.
But since the introduction of the gene therapy in 2011, Daniel no longer suffers from any symptoms and the doctors believe he is cured.
Treatment involves taking bone marrow stem cells - the types of cells that make new blood cells - and replacing the faulty gene code with healthy genes When returned to the patient's body, these hormones begin to produce healthy and disease-free blood cells.




7 children from 8 months to 15 years of age were selected because a suitable bone marrow could not be found. Without a marrow transplant, a patient usually cannot survive puberty.
All patients had eczema and associated recurrent infections and the majority also experienced severe bleeding and autoimmune disease, one of whom even had to be in a wheelchair.
Over the two years following treatment, the patients have progressed from an average of 25 days to no hospital stay. And the baby must be in a wheelchair to be able to walk by himself.
"This is the first time that genetically modified stem cell-based gene therapy has been tested in an international clinical trial," said Fulvio Mavilio, Chief Scientist at Genethon, the biotechnology company that developed the therapy. shows strong and consistent treatment effects in many different settings and in many different countries "
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According to the Telegraph
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