RNA therapy

I01 QRX-704, a novel antisense oligonucleotide therapy, designed to prevent hd pathology while maintaining htt function
Pontus Klein, Zhana Karneva, Lodewijk Toonen, Hyeongju Kim, Nicholas Caron, Linda van der Graaf, Levi Buil, Geert van der Horst, Herma Anthonijsz, Frits van der Ham, Wouter Beumer, Michael Hayden, Ji-Joon Song, Willeke van Roon-Mom, Gerard Platenburg

Journal of Neurology, Neurosurgery & Psychiatry Sep 2018, 89 (Suppl 1) A88; DOI: 10.1136/jnnp-2018-EHDN.237

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RNA therapy