{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T08:33:44Z","timestamp":1740126824479,"version":"3.37.3"},"reference-count":31,"publisher":"Georg Thieme Verlag KG","issue":"04","license":[{"start":{"date-parts":[[2023,4,11]],"date-time":"2023-04-11T00:00:00Z","timestamp":1681171200000},"content-version":"vor","delay-in-days":10,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Fortschr Neurol Psychiatr"],"published-print":{"date-parts":[[2023,4]]},"abstract":"<jats:title>Zusammenfassung<\/jats:title><jats:p>Deutsch: Als h\u00e4ufige genetisch bedingte neurodegenerative Erkrankung ist die Huntington-Krankheit eine Modellerkrankung \u2013 auch f\u00fcr die Gentherapie. Unter den unterschiedlichen M\u00f6glichkeiten ist die Entwicklung von Antisense-Oligonukleotiden am weitesten fortgeschritten. Als weitere Optionen auf Ebene der RNA stehen Mikro-RNAs und Modulatoren der RNA-Prozessierung (Splei\u00dfen) zur Verf\u00fcgung, auf DNA-Ebene Zink-Finger-Proteine. Mehrere Produkte befinden sich in der klinischen Pr\u00fcfung. Diese unterscheiden sich in Applikationsform und systemischer Verf\u00fcgbarkeit, aber auch in der genauen Wirkung. Ein wichtiger Unterschied k\u00f6nnte darin liegen, ob alle Formen des Huntingtin-Proteins gleicherma\u00dfen von der Therapie angesprochen werden, oder ob sich die Therapie pr\u00e4ferentiell gegen besonders toxische Formen wie das Exon1-Protein richtet. Die Ergebnisse der k\u00fcrzlich abgebrochenen GENERATION HD1 Studie waren etwas ern\u00fcchternd, am ehesten aufgrund der nebenwirkungsbedingten Liquorzirkulationsst\u00f6rung. Sie sind daher nur ein Schritt in der Entwicklung zu einer wirksamen Gentherapie gegen die Huntington-Krankheit.<\/jats:p>","DOI":"10.1055\/a-2042-2338","type":"journal-article","created":{"date-parts":[[2023,4,11]],"date-time":"2023-04-11T22:49:04Z","timestamp":1681253344000},"page":"141-146","source":"Crossref","is-referenced-by-count":2,"title":["Gentherapie der Huntington-Krankheit"],"prefix":"10.1055","volume":"91","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2449-5530","authenticated-orcid":false,"given":"Stefan","family":"Br\u00e4uer","sequence":"additional","affiliation":[{"name":"Klinik und Poliklinik f\u00fcr Neurologie, Universit\u00e4tsklinikum an der TU Dresden, Dresden, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2387-526X","authenticated-orcid":false,"given":"Bj\u00f6rn","family":"Falkenburger","sequence":"additional","affiliation":[{"name":"Klinik und Poliklinik f\u00fcr Neurologie, 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