Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

doi:10.1089/hum.2014.109

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Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

Human gene therapy - 1 Mar 2015

Ferla Rita, Claudiani Pamela, Savarese Marco, Kozarsky Karen, Parini Rossella, Scarpa Maurizio, Donati Maria Alice, Sorge Giovanni, Hopwood John J, Parenti Giancarlo, Fecarotta Simona, Nigro Vincenzo, Sivri Hatice Serap, Van Der Ploeg Ans, Andria Generoso, Brunetti-Pierri Nicola, Auricchio Alberto

Abstract excerpt

Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of gene therapy. In the prospect of an AAV8-mediated, liver-directed gene therapy clinical trial for...

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PMID: 25654180
DOI: 10.1089/hum.2014.109
PMCID: PMC4367235

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