antisense oligonucleotides

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
J03 Partial lowering of total huntingtin levels to treat adults with huntington’s disease (hd): potential benefits and theoretical risks from human studies and animal models
Blair R Leavitt, Scott A Schobel, Holly Kordasiewicz

Journal of Neurology, Neurosurgery & Psychiatry Sep 2018, 89 (Suppl 1) A98; DOI: 10.1136/jnnp-2018-EHDN.263
A6 RNA targeted therapies – the experience from SMA and Duchenne’s muscular dystrophy
Francesco Muntoni

Journal of Neurology, Neurosurgery & Psychiatry Sep 2016, 87 (Suppl 1) A2; DOI: 10.1136/jnnp-2016-314597.6
M02 Therapeutic Benefit Of A Htt-lowering Antisense Oligonucleotide Targeting The Cag-repeat In The R6/2 Huntington’s Disease Mouse Model
N Datson, S Mulders, J van de Giessen, A Gonzalez, J van Deutekom

Journal of Neurology, Neurosurgery & Psychiatry Sep 2014, 85 (Suppl 1) A94-A95; DOI: 10.1136/jnnp-2014-309032.274
M03 Complete Huntingtin Haplotypes For Allele-specific Silencing
C Kay, J Collins, N Skotte, A Southwell, A DiPardo, C Ross, F Squitieri, M Hayden

Journal of Neurology, Neurosurgery & Psychiatry Sep 2014, 85 (Suppl 1) A95; DOI: 10.1136/jnnp-2014-309032.275

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antisense oligonucleotides