What does this research mean for the field?

Hybrid AAV2/5 vectors demonstrate greater muscle-directed transduction efficacy than AAV2 vectors without cross-neutralization, and healthy humans show no pre-existing immunity to AAV5 capsids. Novelty: ClaimNovelty.NOVEL_FINDING. Consensus alignment: ConsensusAlignment.NEUTRAL.

July 1, 2001Journal of Virology207 citationsOpen Access

Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer

MHMarkus HildingerThe Wistar Institute AAAlberto AuricchioFederico II University Hospital GGGuangping GaoThe Wistar Institute

Structured PICO

Does AAV2/5 hybrid vector improve muscle-directed gene transfer transduction efficacy compared to AAV2 in immune-competent mice?

Population

Immune-competent mice and a large population of healthy human subjects

Intervention

AAV2/5 hybrid vectors (AAV2 ITRs and Rep, AAV5 Cap) for muscle-directed gene transfer

Comparator

AAV2 vectors

Outcome

Transduction efficacy and cross-neutralizationsurrogate

AAV2/5 hybrid vectors demonstrate superior muscle-directed gene transfer efficacy compared to AAV2 in mice, and humans lack pre-existing immunity to AAV5 capsids, suggesting potential for clinical gene therapy.

Abstract

Vectors based on hybrids consisting of adeno-associated virus types 2 (ITRs and Rep) and 5 (Cap) were evaluated for muscle-directed gene transfer (called AAV2/5). Evaluation in immune-competent mice revealed greater transduction efficacy with AAV2/5 than with AAV2 and no cross-neutralization between AAV2/5 and AAV2. Interestingly, we saw no immunologic evidence of previous exposure to AAV5 capsids in a large population of healthy human subjects.

Ask AI

Helpful

Bookmark

View Full Paper