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

Willeke M C van Roon-Mom1, Raymund A C Roos2, Susanne T de Bot2

  • 11 Department of Human Genetics, Leiden University Medical Center , Leiden, the Netherlands .

Nucleic Acid Therapeutics
|April 6, 2018
PubMed

Keywords:
Huntington diseaseclinical trialpatients

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