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rAAV-Based Compositions and Methods for Treating Alpha-1 Antitrypsin Related Conditions

Detailed Technology Description: 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

Countries: Not Available

Application No.: None

*Abstract: 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 protein (AAT), and 2) gene for modified and functional AAT protein that is not targeted by the aforementioned miRNA.

*IP Issue Date: None

*IP Type: Other Patent

Country/Region: USA

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