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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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Bibliographic Details
Published in:Pharmaceuticals (Basel)
Main Authors: Tickner, Zachary J., Farzan, Michael
Format: Artigo
Language:Inglês
Published: MDPI 2021
Subjects:
Online Access: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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