Author

T. Cornu

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#gene editing Open access Sep 2026

Targeted insertion of an optimized donor DNA is effective in a humanized mouse model of dominant retinitis pigmentosa.

Retinitis pigmentosa (RP) affects 1 in 3,000 individuals worldwide, with 30%-40% of cases inherited as autosomal dominant (AD). Mutations in RHO (RP4) are the most common cause of ADRP. Because most RHO mutations exert gain-of-function or dominant-negative effects, conventional gene supplementation is insufficient, req...

F. Esposito, Arjun Padmanabhan, M. Rhiel et al. · Cell Reports Medicine · 0 citations

Review Aug 2026

Accelerating Translation of NK Cell Therapies to the Clinic: A European Perspective

Addressing challenges facing the clinical translation of NK cell therapies was one of the issues discussed in the NK & ILC Symposium that took place in Freiburg, Germany from March 11 to 13, 2026, as the annual meeting of the NK & ILC study group of the German Society for Immunology.

L. Knapp, Katharina Sophie Fischer, Janika Sosat et al. · Human Gene Therapy · 0 citations

Open access Jul 2026

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model

The results suggest that this approach may overcome the reliance on busulfan or other myeloablative conditioning regimens with their associated morbidities, and by enabling toxin-free conditioning and in vivo selection of edited cells, may facilitate clinical implementation of these highly valuable genetic therapies.

Romina Marone, Rosalba Lepore, K. Paschoudi et al. · bioRxiv · 0 citations