Gene therapy trial aims to fix protein deficiency at its source
NCT ID NCT07227207
Summary
This early-stage study is testing a new gene therapy called TSRA-196 for adults with severe Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can damage the lungs and liver. The main goals are to find a safe dose and see if the treatment can raise the body's levels of a crucial protective protein to a normal range. The study will enroll about 72 people who have either lung or liver problems caused by the disease.
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Conditions
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