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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....
Source: reuters.com