Targeting Huntingtin Expression in Patients with Huntington’s Disease

Autor: Tabrizi, Sarah J, Leavitt, Blair R, Rosser, Anne, Kordasiewicz, Holly B, Czech, Christian, Swayze, Eric E, Norris, Daniel A, Baumann, Tiffany, Gerlach, Irene, Schobel, Scott A, Paz, Erika, Smith, Anne V, Landwehrmeyer, G Bernhard, Bennett, C Frank, Lane, Roger M, Teams, Phase 1–2a IONIS-HTTRx Study Site, McColgan, Peter, Hensman, Davina, Ghosh, Rhia, Flower, Michael, Libri, Vincenzo, Saunders, Edwina, Raymond, Lynn, Wild, Edward J, Decolongon, Joji, Li, Tuan, Fathinia, Panteha, Lang, Christina, Lewerenz, Jan, Lindenberg, Katrin, Ludolph, Albert C, Schneider, Ariane, Trautmann, Sonja, Uhl, Stefanie, Saft, Carsten, Weydt, Patrick, Kaminski, Barbara, Kaminski, Daniela, Hoffmann, Rainer, von Hein, Sarah M, Muhlack, Siegfried, Gold, Ralf, Collins, Lucy, Mason, Sarah, Scott, Kirsten, Barker, Roger A, Stoker, Tom, Greenland, Julia, Andresen, Katie, Shanmugam, Mohan, Abdelghani, Mowafak, Turgut, Tolga, Peeren, Siofra, Colaco, Olga, Owens, Rebecca, Subin, Sujamole, Blair, Nick F, Spruth, Eike Jakob, Beckmann, Janna, Krug, Henriette, Langenfurth, Anika, Crossley, Diana, Akhtar, Nasreen, Gavin, Jennie, De Souza, Jenny, Massey, Thomas, McLauchlan, Duncan, Craufurd, David, Cousins, Rebecca, Shastin, Dmitri, Peall, Kathryn, Bhatt, Harsh, Davison, Andrew, Bagshawe, Joanne, Gunning, Belinda, Hamandi, Khalid, Priller, Josef, Rickards, Hugh

Publikováno v: 2019, ' Targeting Huntingtin Expression in Patients with Huntington’s Disease ', New England Journal of Medicine, vol. 380, no. 24, pp. 2307-2316 . https://doi.org/10.1056/NEJMoa1900907
The New England journal of medicine 380(24), 2307-2316 (2019). doi:10.1056/NEJMoa1900907

BACKGROUNDHuntington’s disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT, resulting in a mutant huntingtin protein. IONIS-HTTRx (hereafter, HTTRx) is an antisense oligonucleotide designed

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ac9b0c3cfd83bb7bd722e3850edd8afa
https://www.pure.ed.ac.uk/ws/files/89496225/nejmoa1900907.pdf