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Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll

13 Jul 2020

Abstract excerpt

Gene therapy using recombinant adeno-associated virus (rAAV) vectors has demonstrated definitive benefits for genetic diseases and has enormous future potential, representing an important part of the next paradigm of human therapeutics. However, many clinical trials with rAAV have reported varying degrees of immunotoxicity, potentially including that associated with the recently reported deaths of two subjects in...

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PMID: 32710825
DOI: 10.1016/j.ymthe.2020.07.006

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