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Article

Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles

3 May 2018

Abstract excerpt

The Adeno-associated virus (AAV) gene delivery system is ushering in a new and exciting era in the United States; following the first approved gene therapy (Glybera) in Europe, the FDA has approved a second therapy, Luxturna [1].However, challenges to this system remain.In viral gene therapy, the surface of the capsid is an important determinant of tissue tropism, impacts gene transfer efficiency, and is targeted...

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PMID: 29723270
DOI: 10.1371/journal.ppat.1006929

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