Treating Pigs With A Gene Therapy For Huntington’s Disease
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Our mission at uniQure is to deliver curative gene therapies that transform the lives of patients. We have an ongoing clinical program in hemophilia B and preclinical proof-of-concept for gene therapies in Huntington's disease, spinocerebellar ataxia type 3, hemophilia A, and Fabry.

                       

Treating Pigs With A Gene Therapy For Huntington’s Disease

Back in 1993, a gene was discovered to be responsible for the heritable brain disorder called Huntington’s disease. It was found that within the gene called huntingtin (HTT), a repetitive piece of coding sequence was abnormally expanded, inevitably causing Huntington’s disease. If one of your parents has the disease-causing mutation, you have a fifty percent chance of getting Huntington’s disease. This repeat expansion in the HTT gene is translated in a mutant huntingtin protein that acquires toxic properties. The toxic...

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