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T. Cantz

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#gene editing Case report Open access Nov 2026

Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo Base Editing in a Patient with Alpha-1 Antitrypsin Deficiency.

Sustained biologically active AAT levels support feasibility and potential of this approach for the treatment of AATD, and substantial and precise hepatic correction of the pathogenic SERPINA1 PI*ZZ variant is achieved.

S. Krooss, Tai-Hua Yang, Q. Yuan et al. · 0 citations
Editorial Open access Jul 2026

The German National Strategy for Gene- and Cell-Based Therapies: Generating Impact by Employing a Novel Multi-Stakeholder Approach

The main goal of the German National Strategy for GCT is to ensure patient access to advanced therapies while strengthening Germany’s position as an international hub for biomedical innovation.

Christian Gallus, F. Ayuk, P. Beckhove et al. · 0 citations

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