Author

H. Büning

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Review Open access Jan 2026

Insights Into RP2‐Associated Retinopathy: From Molecular Basis to Clinical Translation

Pathogenic variants in RP2 cause X‐linked RP2‐associated retinopathy, a rare inherited retinal disorder typically characterized by childhood onset and progression to severe visual impairment within the first three decades of life. Despite its clinical severity, comprehensive clinical and genetic data on RP2‐associated...

N. Bögershausen, Patricia Döring, Maximilian J. Gerhardt et al. · Human Mutation · 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. · Human Gene Therapy · 0 citations

Jul 2026

Dichotomies in Ex Vivo and In Vivo Performance of Receptor-Binding Mutants of Adeno-Associated Virus Vectors

In vivo performance diverged sharply from ex vivo findings as AAV2ΔHSPG and AAV2ΔHSPG and AAV2ΔHSPGΔIntegrin observed a substantially enhanced transduction in multiple non-hepatic tissues, as well as a strong liver detargeting, while AAV2ΔIntegrin was non-infectious in vivo.

Olaniyi Olarewaju, Martin Bentler, Jacqueline Breuer et al. · Human Gene Therapy · 0 citations