rAAV-Based Compositions and Methods for Treating Alpha-1 Antitrypsin Related Conditions
- 详细技术说明
- 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
- *Abstract
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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
- 国家
- Not Available
- 申请号码
- None
- 国家/地区
- 美国
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