Population
In vitro and in vivo muscle models
Comparison
AAV capsid mutants vs Wild-type AAV1 and AAV2 vectors
Design
Preclinical
Authors
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Hypothesis-generating for muscle-targeted AAV capsid engineering; requires validation in larger models before clinical translation.
Amino acids 350 to 430 of the AAV1 VP1 capsid protein are a major determinant for efficient muscle transduction, which may inform future gene delivery vector design.
Hauck et al. (2003) studied this question.
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