Examinando por Autor "Sánchez Gutiérrez, Cristina"

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    Pre-existing antibodies to gene therapy AAV vectors in colombian healthy donors and patients with neurodegenerative diseases: implications for clinical translation
    (Universidad de los Andes, 2025-12-04) Sánchez Gutiérrez, Cristina; Valderrama Aguirre, Augusto Elias; Rincón Acelas, Melvin Yesid; Lattig Matiz, María Claudia; González Aseguinolaza, Gloria
    Adeno-associated virus (AAV) vectors are widely utilized in gene therapy due to their favorable safety profile, low immunogenicity, and organ-specific targeting capabilities. Pre-existing humoral immunity, particularly neutralizing antibodies (NAbs), can impede vector entry and therapeutic efficacy, while total antibodies (TAbs) or non-neutralizing antibodies may alter vector biodistribution. Seroepidemiological data from Latin America, particularly Colombia, remain scarce. This study aimed to (i) perform a global scoping review of TAb and NAb prevalence, (ii) assess TAbs against AAV2 and AAV9 in Colombian healthy donors and patients with neurological diseases, and (iii) conduct a pilot NAb analysis. The scoping review followed PRISMA guidelines, systematically identifying peer-reviewed studies reporting antibody prevalence against AAV across diverse populations. Concurrently, sera from defined Colombian cohorts were analyzed, with TAbs quantified via ELISA against AAV2 and AAV9, and NAbs evaluated using a transduction inhibition assay in a pilot study of healthy donors. Results showed high seroprevalence of anti-AAV2 antibodies across all groups, with lower prevalence for AAV9, although the neurological cohort exhibited elevated levels for both serotypes. Observed seroprevalence patterns align with international reports despite demographic and methodological differences. This study provides a comprehensive characterization of AAV immunity in Colombia, establishing a framework to guide future surveillance, support patient stratification, and inform the rational design of AAV-based therapeutic strategies in the region.