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Article

Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?

8 Feb 2020

Abstract excerpt

Recombinant AAV (rAAV) vectors are a key component of an emergent therapeutic paradigm with a demonstrated definitive benefit for genetic diseases. Immune responses are among the most challenging barriers to human gene therapy. Viral vectors are highly advantageous because of their evolved ability to negotiate complex intracellular pathways to efficiently deliver a DNA payload; however, the human immune system...

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PMID: 32035026
DOI: 10.1016/j.ymthe.2020.01.026

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