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Gene Therapy for Monogenic Inherited Disorders: Opportunities and Challenges

BACKGROUND: New gene therapy approaches have emerged as promising treatment options for rare congenital disorders and certain tumor entities for which previously only procedures of limited curative potential had been available, if at all. METHODS: Based on a selective literature search, the principl...

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Publicat a:Dtsch Arztebl Int
Autors principals: Kirschner, Janbernd, Cathomen, Toni
Format: Artigo
Idioma:Inglês
Publicat: Deutscher Arzte Verlag 2020
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8045130/
https://ncbi.nlm.nih.gov/pubmed/33637169
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3238/arztebl.2020.0878
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