Key result
rAAV1 vector mediated at least 100-fold higher levels of functional human alpha 1 antitrypsin secretion in mouse muscle compared to the rAAV2 vector.
Population
Mouse muscle model
Comparison
Recombinant adeno-associated virus 1 vector for… vs Other rAAV serotypes (rAAV2, 3, 4, and 5)
Design
Preclinical
Authors
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Supports advancing rAAV1 to human AAT trials; animal data leave clinical translation open.
rAAV1 is a highly efficient vector for muscle-directed AAT gene therapy that produces functional hAAT, supporting its potential use in clinical trials.
Lu et al. (2006) studied Alpha 1 antitrypsin deficiency. rAAV1 vector vs. rAAV2 vector was evaluated on Transduction efficiency and AAT secretion. rAAV1 vector mediated at least 100-fold higher levels of functional human alpha 1 antitrypsin secretion in mouse muscle compared to the rAAV2 vector.
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