Why the study?
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
Comparison
AAV2/5 hybrid vectors for muscle-directed gene… vs AAV2 vectors
Design
Preclinical
Authors
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May enhance muscle gene transfer in immune-competent models; leaves open clinical translation of AAV2/5.
Does AAV2/5 hybrid vector improve muscle-directed gene transfer transduction efficacy compared to AAV2 in immune-competent mice?
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.
Hildinger et al. (2001) studied this question.
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