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Tuesday, March 7, 2017

First sickle cell patient gets cured with gene therapy

 

This is indeed good news for the world and especially for those living with sickle cell anaemia. 

A 13-year-old has been cured of sickle cell anemia with the aid of gene therapy which allowed complete remission of clinical symptoms of disease and correction of biological signs. 

Gene therapy is the introduction of normal genes into cells in place of defective or missing genes in order to correct genetic disorders.

According to the result of a study published in the New England Journal of Medicine, when the gene therapy is fully deployed, the patient would no longer have to battle the complications and pain resulting from the disease.

A team led by Pr. Marina Cavazzana in collaboration with Dr. Philippe Leboulch in October 2014 carried out gene therapy in the context of a phase I clinical trial/II in a 13-year-old patient with severe sickle cell disease. 

In September, scientists reported that gene therapy for sickle cell disease passed key preclinical test.



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