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Article

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

1 Jan 2014

Abstract excerpt

Gene therapy strategies for congenital myopathies may require repeat administration of adeno-associated viral (AAV) vectors in response to several limitations inherent to the clinical design: 1) administration of doses below therapeutic efficacy in patients enrolled in early phase clinical trials; 2) progressive reduction of the therapeutic gene expression over time as a result of increasing muscle mass in...

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PMID: 25541616
DOI: 10.1038/mtm.2014.33

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