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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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Detalhes bibliográficos
Publicado no:Dtsch Arztebl Int
Main Authors: Kirschner, Janbernd, Cathomen, Toni
Formato: Artigo
Idioma:Inglês
Publicado em: Deutscher Arzte Verlag 2020
Assuntos:
Acesso em linha: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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