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Riboswitches for Controlled Expression of Therapeutic Transgenes Delivered by Adeno-Associated Viral Vectors

Vectors developed from adeno-associated virus (AAV) are powerful tools for in vivo transgene delivery in both humans and animal models, and several AAV-delivered gene therapies are currently approved for clinical use. However, AAV-mediated gene therapy still faces several challenges, including limit...

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Dades bibliogràfiques
Publicat a:Pharmaceuticals (Basel)
Autors principals: Tickner, Zachary J., Farzan, Michael
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8230432/
https://ncbi.nlm.nih.gov/pubmed/34200913
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ph14060554
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