Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
收藏DataMed2026-07-12 收录
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资源简介：
Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency.
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ClinicalTrials.gov
