{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2024,6,28]],"date-time":"2024-06-28T00:02:57Z","timestamp":1719532977343},"reference-count":100,"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>Die Diagnose von Motoneuronerkrankungen wie Amyotrophe Lateralsklerose (ALS) und\n                    5q-assoziierte Spinale Muskelatrophie (SMA) bedeuteten in der Vergangenheit\n                    schicksalshafte Machtlosigkeit gegen\u00fcber scheinbar unbehandelbaren\n                    Erkrankungen mit schwersten motorisch-funktionellen Einschr\u00e4nkungen und\n                    teils fatalen Krankheitsverl\u00e4ufen. J\u00fcngste Fortschritte im\n                    Verst\u00e4ndnis der genetischen Kausalit\u00e4ten dieser Erkrankungen\n                    kombiniert mit Erfolgen in der Entwicklung von gezielten Gentherapiestrategien\n                    bewirken eine hoffnungsvolle Wende hin zu erstmals effektiven, innovativen\n                    Therapiekonzepten gleichsam als Pionier in der Behandlungsf\u00e4higkeit\n                    neurodegenerativer Erkrankungen. W\u00e4hrend f\u00fcr die SMA seit\n                    wenigen Jahren Gentherapien bereits zugelassen werden konnten, befindet sich die\n                    Gentherapieerforschung bei der ALS mit ermutigenden Resultaten noch in der\n                    klinischen Pr\u00fcfung. Dieser Artikel gibt einen \u00dcberblick\n                    \u00fcber die bisher bekannten genetischen Hintergr\u00fcnde von ALS und\n                    SMA sowie deren Gentherapie-Ans\u00e4tze mit Fokus auf Therapiekandidaten,\n                    die sich in klinischen Pr\u00fcfungen befinden oder bereits die Markzulassung\n                    erworben haben.<\/jats:p>","DOI":"10.1055\/a-2002-5215","type":"journal-article","created":{"date-parts":[[2023,2,23]],"date-time":"2023-02-23T23:49:50Z","timestamp":1677196190000},"page":"153-163","source":"Crossref","is-referenced-by-count":0,"title":["Gentherapien bei den Motoneuronerkrankungen ALS und\n                    SMA"],"prefix":"10.1055","volume":"91","author":[{"given":"Ren\u00e9","family":"G\u00fcnther","sequence":"additional","affiliation":[{"name":"Klinik und Poliklinik f\u00fcr Neurologie, University Hospital Carl\n                    Gustav Carus at Technische Universit\u00e4t Dresden, Dresden,\n                    Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2023,2,23]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"540","DOI":"10.1136\/jnnp-2016-315018","article-title":"Genetic epidemiology of amyotrophic lateral sclerosis: a systematic review and\n                    meta-analysis","volume":"88","author":"Z Y Zou","year":"2017","journal-title":"Journal of neurology, neurosurgery, and psychiatry"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1016\/j.ncl.2015.07.004","article-title":"Spinal Muscular Atrophy","volume":"33","author":"S J Kolb","year":"2015","journal-title":"Neurologic clinics"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1136\/jnnp.2009.183525","article-title":"Incidence of amyotrophic lateral sclerosis in Europe","volume":"81","author":"G Logroscino","year":"2010","journal-title":"Journal of neurology, neurosurgery, and 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