Gene therapy successfully corrects genetic defect in alpha-1 antitrypsin deficiency
In a landmark achievement in the field of genetic medicine, researchers have reported the very first published case in a peer-reviewed journal of a successful clinical treatment of a patient with alpha-1 antitrypsin deficiency (PiZZ) using in vivo gene therapy.
- Published by
- News-Medical
- Evidence type
- Clinical
In a landmark achievement in the field of genetic medicine, researchers have reported the very first published case in a peer-reviewed journal of a successful clinical treatment of a patient with alpha-1 antitrypsin deficiency (PiZZ) using in vivo gene therapy.
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