Title : Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities.

Pub. Date : 2020 Oct

PMID : 32796930






1 Functional Relationships(s)
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1 Therapeutically, the first human trial of an HTT-lowering antisense oligonucleotide successfully, and safely, reduced the CSF concentration of mHTT in individuals with HD. mhtt huntingtin Homo sapiens