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Early phase drug trial claims a breakthrough in Huntington disease. IONIS-HTT is an antisense "gene-silencing" compound,
Early phase drug trial claims a breakthrough in Huntington disease. IONIS-HTT is an antisense "gene-silencing" compound, injected in to CSF and designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for Huntington's Disease. The trial has not been reported at a scientific conference or submitted for peer review but made global news today. Hope for its use in a asymptomatic carriers of the autosomal dominant gene will drive further study. Roche has exercised its option to take over the drug development.
"'Ground-breaking' new drug gives hope in Huntington's disease: Scientists have for the first time fixed a protein defect that causes Huntington's disease by injecting a drug from Ionis Pharmaceuticals (IONS.O) into the spine, offering new hope for patients with the devastating genetic disease."
Source: uk.reuters.com
huntington disease drug hope protein trial autosomal carriers
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