Author

Toni Cathomen

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#gene editing Open access Sep 2026

p38 MAPK inhibition safeguards genome integrity during gene editing of hematopoietic stem and progenitor cells.

Ex vivo genome editing of hematopoietic stem and progenitor cells (HSPCs) holds significant therapeutic potential but remains constrained by genotoxic risks associated with nuclease-induced DNA double-strand breaks, DNA donor template delivery and sensing, and proliferation-induced stress during ex vivo manipulation. T...

Roberta Vacca, Lucrezia della Volpe, Nicolò Gualandi et al. · Molecular Therapy · 0 citations

#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

Open access Jul 2026

Systematic Benchmarking of CRISPR-Cas9 Off-Target Prediction Tools Reveals Limitations and Implications for Preclinical Assessment.

Accurate identification of CRISPR-Cas9 off-target sites is essential for the safety assessment of genome-editing-based therapies. While numerous in silico prediction tools have been developed, their comparative performance and practical utility in preclinical workflows remain incompletely defined. We performed a system...

M. M. Kaufmann, Maren Hackenberg, William Jobson Pargeter 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

Open access Jul 2026

Base editing-derived models of human WDR34 and WDR60 disease alleles replicate retrograde intraflagellar transport (IFT) and hedgehog signaling defects

Base editing derived cell models shed light on the role of ciliary dynein complex components for intraflagellar transport, hedgehog signaling and differential regulation of genes associated with Golgi intracellular transport using human disease alleles.

Dinu Antony, Elif Yilmaz Güleç, A. Klawonn et al. · Communications Biology · 0 citations