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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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Bibliografische gegevens
Gepubliceerd in:Dtsch Arztebl Int
Hoofdauteurs: Kirschner, Janbernd, Cathomen, Toni
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Deutscher Arzte Verlag 2020
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Online toegang: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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