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Wichtige Voraussetzungen f\u00fcr eine fr\u00fch im Krankheitsprozess einsetzende und deshalb den weiteren Verlauf der Krankheit in klinisch relevanter Weise modifizierende Therapie sind damit gegeben und machen die HK zu einer Modellerkrankung f\u00fcr neuroprotektive Behandlungsans\u00e4tze. In der Vergangenheit lag der Schwerpunkt auf dem Ausgleich vermuteter Neurotransmitterdefizite (GABA) analog zur Parkinson-Erkrankung und auf klassischen neuroprotektiven Strategien zur Beeinflussung hypothetischer gemeinsamer Endstrecken neurodegenerativer Erkrankungen (z.\u202fB. Exzitotoxizit\u00e4t, mitochondriale Dysfunktion, oxidativer Stress etc.). Mit der Entdeckung der krankheitsverursachenden<jats:italic>HTT<\/jats:italic>-Mutation im Jahr 1993 fokussierte sich die Therapieforschung zunehmend darauf, soweit proximal wie m\u00f6glich in die pathophysiologische Ereigniskette einzugreifen. Ein wichtiger Ansatzpunkt ist hier die<jats:italic>HTT<\/jats:italic>-mRNA mit dem Ziel, die Nachproduktion mutierter Huntingtin-Genprodukte zu senken und damit den K\u00f6rper von deren sch\u00e4digenden Auswirkungen zu entlasten; zu diesem Zweck sind verschiedene Behandlungsmodalit\u00e4ten (einzelstr\u00e4ngige DNA und RNA, divalente RNA und Zinkfinger-Repressorkomplexe, oral verf\u00fcgbare Splei\u00dfmodulatoren) entwickelt worden, die sich in der klinischen Pr\u00fcfung (Phase\u00a0I\u2013III) oder in sp\u00e4ten Stadien der pr\u00e4klinischen Entwicklung befinden. Zudem zeichnet sich ab, dass es m\u00f6glich sein k\u00f6nnte, die L\u00e4nge der somatisch instabilen, d.\u202fh. \u00fcber die Lebenszeit v.\u202fa. im Hirngewebe zunehmende CAG-Mutation selbst zu beeinflussen und die Progression der HK hierdurch zu bremsen.<\/jats:p>","DOI":"10.1007\/s00115-021-01224-8","type":"journal-article","created":{"date-parts":[[2021,11,11]],"date-time":"2021-11-11T16:03:10Z","timestamp":1636646590000},"page":"179-190","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Krankheitsmodifizierende Therapieans\u00e4tze bei der Huntington-Krankheit","Disease-modifying treatment approaches in Huntington disease"],"prefix":"10.1007","volume":"93","author":[{"given":"Wiebke","family":"Frank","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katrin S.","family":"Lindenberg","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alzbeta","family":"M\u00fchlb\u00e4ck","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jan","family":"Lewerenz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"G. 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W.\u00a0Frank gibt an, dass kein Interessenkonflikt besteht. K.S.\u00a0Lindenberg gibt an, dass ihre Institution f\u00fcr ihre Rolle als Hauptpr\u00fcferin in Studien f\u00fcr Prilenia und der Cure Huntington\u2019s Disease Initiative (CHDI) Foundation verg\u00fctet wurde. A.\u00a0M\u00fchlb\u00e4ck gibt an, dass ihre Institution f\u00fcr ihre Rolle als Hauptpr\u00fcferin in Studien f\u00fcr Prilenia, CHDI und Wave verg\u00fctet wurde. J.\u00a0Lewerenz erhielt Vortragshonorare oder Reisekostenerstattungen von UCB, Bayer, Hoffmann-LaRoche, TEVA und der CHDI Foundation. Seine Institution wurde f\u00fcr seine Rolle als Hauptpr\u00fcfer in Studien f\u00fcr UCB und CHDI verg\u00fctet. Seine Forschung wird von der Europ\u00e4ischen Huntington-Initiative und dem Ministerium f\u00fcr Bildung und Forschung Baden-W\u00fcrttemberg und dem Bundesministerium f\u00fcr Bildung und Forschung (BMBF) finanziert. G.B.\u00a0Landwehrmeyer ist im Scientifc Advisory Board f\u00fcr Hoffmann-LaRoche, Novartis, PTC Therapeutics, Remix, Takeda, TEVA und Triplet Therapeutics. Zudem ist er als Berater f\u00fcr Acadia Pharm, AOP Orphan, Boehringer-Ingelheim, CHDI Foundation, Genetech, Hoffmann-LaRoche, Ionis Pharma, Lundbeck, NeuraMetrix, Novartis, Prilenia, PTC Therapeutics, TEVA, Triplet Therapeutics, Takeda und Wave t\u00e4tig und erhielt Honorare von Genetech, Hoffmann-LaRoche, Novartis und Teva Pharmaceuticals. Seine Institution, das Huntington-Zentrum der Abteilung Neurologie der Universit\u00e4t Ulm, hat als Kompensation f\u00fcr klinische Studien mit Hoffmann-LaRoche, Ionis-Pharma, Prilenia, TEVA Zuwendungen erhalten. Das Europ\u00e4ische HD Netzwerk (EHDN) hat als Kompensation f\u00fcr klinische Beratung von Hoffmann-LaRoche und TEVA Zuwendungen erhalten. Seine Forschung wird von der CHDI Foundation, der EU (FP6&7, Horizon2020, JPND), dem BMBF und der DFG finanziert.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Interessenkonflikt"}},{"value":"F\u00fcr diesen Beitrag wurden von den Autoren keine Studien an Menschen oder Tieren durchgef\u00fchrt. F\u00fcr die aufgef\u00fchrten Studien gelten die jeweils dort angegebenen ethischen Richtlinien.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Interessenkonflikt"}}]}}