Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Jct van Deutekom"'
Autor:
Annemieke Aartsma-Rus, Gjb van Ommen, Sam Mulders, J.T. den Dunnen, Melvin M. Evers, Hoang-Dai Tran, Wmc van Roon-Mom, Jct van Deutekom
Publikováno v:
Journal of Neurology, Neurosurgery & Psychiatry. 83:A52.1-A52
There has been a recent surge in research using antisense oligonucleotides (AONs) to reduce huntingtin transcript levels and thus huntingtin protein levels both in vitro an in vivo. This can be done in a non-allele specific manner by targeting both m
Autor:
Jct van Deutekom, J van de Giessen, Sam Mulders, A Jahanshahianvar, Yasin Temel, Rinske Vlamings
Publikováno v:
Journal of Neurology, Neurosurgery & Psychiatry. 83:A52.2-A52
Background Huntington9s disease (HD) is caused by an expanded (CAG)n tract in the Huntingtin gene ( HTT) that is translated into an expanded polyglutamine (polyQ) stretch in the protein. The polyQ stretch causes a toxic gain of function and plays a c
Autor:
Kourkouta E; BioMarin Nederland BV, Leiden, the Netherlands., Weij R; BioMarin Nederland BV, Leiden, the Netherlands., González-Barriga A; BioMarin Nederland BV, Leiden, the Netherlands., Mulder M; BioMarin Nederland BV, Leiden, the Netherlands., Verheul R; BioMarin Nederland BV, Leiden, the Netherlands., Bosgra S; BioMarin Nederland BV, Leiden, the Netherlands., Groenendaal B; BioMarin Nederland BV, Leiden, the Netherlands., Puoliväli J; Charles River Discovery Research Services, Kuopio, Finland., Toivanen J; Charles River Discovery Research Services, Kuopio, Finland., van Deutekom JCT; BioMarin Nederland BV, Leiden, the Netherlands., Datson NA; BioMarin Nederland BV, Leiden, the Netherlands. Electronic address: nicole.datson@bmrn.com.
Publikováno v:
Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2019 Sep 06; Vol. 17, pp. 601-614. Date of Electronic Publication: 2019 Jul 19.
Autor:
González-Barriga A; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands .; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., Nillessen B; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands ., Kranzen J; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands ., van Kessel IDG; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands ., Croes HJE; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands ., Aguilera B; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., de Visser PC; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., Datson NA; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., Mulders SAM; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., van Deutekom JCT; 2 BioMarin Nederland B.V., Leiden, the Netherlands ., Wieringa B; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands ., Wansink DG; 1 Department of Cell Biology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center , Nijmegen, the Netherlands .
Publikováno v:
Nucleic acid therapeutics [Nucleic Acid Ther] 2017 Jun; Vol. 27 (3), pp. 144-158. Date of Electronic Publication: 2017 Apr 04.