Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT(2023)
摘要
Pre-existing anti-adeno-associated virus (AAV) antibodies can impede successful gene transfer and potentially have safety consequences for patients receiving recombinant AAV (rAAV)-mediated gene therapy (GTx). Hence, assessing patients’ anti-AAV antibody status is an important consideration prior to systemic rAAV GTx administration.
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