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Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM

3 Jun 2022

Abstract excerpt

In spite of continuous development of gene therapy vectors with thousands of drug candidates in clinical drug trials there are only a small number approved on the market today stressing the need to have characterization methods to assist in the validation of the drug development process. The level of packaging of the vector capsids appears to play a critical role in immunogenicity, hence an objective quantitative...

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PMID: 35657790
DOI: 10.1371/journal.pone.0269139

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