This invention uses a dual-specificity AAV vectors to correct Alpha 1-Antitrypsin (AAT) deficiency. This dual vector carries: 1) miRNAs that target and inhibit the expression of the mutant endogenous
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Recognizes the targetprotein when used in various techniques
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• Enhanced transfection efficiency (up to a 30-fold increase) • Customizable to various cell types and conditions including the presence of serum
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· Diminishedseverity of disease· Less toxicside effects
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None
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