Title : Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease Patients.

Pub. Date : 2018 Apr

PMID : 29620999






2 Functional Relationships(s)
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1 Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease Patients. Oligonucleotides huntingtin Homo sapiens
2 The results from this Ionis trial have gained much attention from the patient community and the oligonucleotide therapeutics field, since it is the first trial targeting the cause of HD, namely the mutant huntingtin protein, using antisense oligonucleotides (ASOs). Oligonucleotides huntingtin Homo sapiens