{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,15]],"date-time":"2025-12-15T19:50:54Z","timestamp":1765828254330,"version":"3.37.3"},"reference-count":34,"publisher":"Georg Thieme Verlag KG","issue":"04","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>Ataxien sind progredient verlaufende Krankheiten, die meist Folge einer\n                    Degeneration des Kleinhirns sind. Ataxien werden in genetische, sporadisch\n                    degenerative und erworbene (sekund\u00e4re) Formen unterteilt.\n                    W\u00e4hrend es bei den erworbene (sekund\u00e4re) Ataxien etablierte\n                    Therapien gibt, sind genetische und sporadische degenerative Ataxien derzeit\n                    nicht medizinisch behandelbar. F\u00fcr diese Ataxien ist die Entwicklung\n                    somatischer Gentherapien ein vielversprechender Weg. Ziele der Gentherapien bei\n                    genetischen Ataxien sind die Inaktivierung sch\u00e4dlicher Gene durch\n                    Gen-Silencing oder der Ersatz oder die Korrektur eines nicht\n                    funktionsf\u00e4higen Gens. Eine weitere Option, die auch f\u00fcr\n                    sporadisch degenerative Ataxien in Betracht kommt, sind Therapien, bei denen\n                    neue oder modifizierte Gene transferiert werden. Bei den h\u00e4ufigeren\n                    Ataxien, wie Friedreich-Ataxie, bestimmten spinozerebell\u00e4ren Ataxien und\n                    Multisystematrophie werden aktiv Gentherapien entwickelt, und erste Phase\n                    I-Studien werden bereits durchgef\u00fchrt.<\/jats:p>","DOI":"10.1055\/a-2015-3305","type":"journal-article","created":{"date-parts":[[2023,2,21]],"date-time":"2023-02-21T00:06:33Z","timestamp":1676937993000},"page":"147-152","source":"Crossref","is-referenced-by-count":2,"title":["Gentherapie f\u00fcr Ataxien"],"prefix":"10.1055","volume":"91","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6174-5442","authenticated-orcid":false,"given":"Thomas","family":"Klockgether","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Neurologie, Universit\u00e4tsklinikum Bonn und\n                    Deutsches Zentrum f\u00fcr Neurodegenerative Erkrankungen (DZNE),\n                    Bonn"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2023,2,20]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"560","DOI":"10.1016\/j.neuron.2019.01.049","article-title":"Autosomal Recessive Cerebellar Ataxias: Paving the Way toward Targeted Molecular\n                    Therapies","volume":"101","author":"M Synofzik","year":"2019","journal-title":"Neuron"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1038\/s41572-019-0074-3","article-title":"Spinocerebellar ataxia","volume":"5","author":"T Klockgether","year":"2019","journal-title":"Nat. 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