Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay | Zendy

Karina Krotova | Zendy; George Aslanidi | Zendy

Open Access

Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay

Author(s) -

Karina Krotova,

George Aslanidi

Publication year - 2020

Publication title -

human gene therapy

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.633

H-Index - 149

eISSN - 1557-7422

pISSN - 1043-0342

DOI - 10.1089/hum.2020.074

Subject(s) - adeno associated virus , genetic enhancement , virology , serotype , antibody , medicine , neutralizing antibody , population , virus , vector (molecular biology) , gene , biology , immunology , genetics , environmental health , recombinant dna

Adeno-associated virus (AAV)-based gene therapy is undergoing major expansion into clinical practice, with two treatments currently being granted Food and Drug Administration (FDA) approval. However, the presence of pre-existing neutralizing antibodies (NAB) is one of the significant hurdles for the clinical application of AAV vectors that significantly limits the patient population, which benefits from the treatment. A reliable diagnostic to evaluate the patient's seropositivity is required to ensure the effectiveness of the AAV-mediated therapeutic. Here, we describe a simple method for the determination of AAV NAB activity based on our finding that Compound C makes HEK293 cell highly permissive for infection by 10 commonly used AAV serotypes.

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