Scientific articles

Kalter, Nechama, et al. "Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges". https://doi.org/10.1016/j.omtn.2025.102636

Cullot, Grégoire, et al. « Genome Editing with the HDR-Enhancing DNA-PKcs Inhibitor AZD7648 Causes Large-Scale Genomic Alterations ». Nature Biotechnology, vol. 43, [...]

Venkatesan, Vigneshwaran, et al. « Editing the Core Region in HPFH Deletions Alters Fetal and Adult Globin Expression for Treatment of [...]

Dacquay, Louis C., et al. « Dual Inhibition of DNA-PK and Polϴ Boosts Precision of Diverse Prime Editing Systems ». Nature Communications, [...]

Sobrino, Steicy, et al. « Severe Inflammation and Lineage Skewing Are Associated with Poor Engraftment of Engineered Hematopoietic Stem Cells in [...]

Brusson, Megane, et Annarita Miccio. « Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies ». médecine/sciences, vol. 41, no 1, janvier 2025, p. [...]

Frati, Giacomo, et al. « Safety and Efficacy Studies of CRISPR-Cas9 Treatment of Sickle Cell Disease Highlights Disease-Specific Responses ». Molecular Therapy: [...]

Wang, Jinglong, et al. « DNA-PKcs Suppresses Illegitimate Chromosome Rearrangements ». Nucleic Acids Research, février 2024, p. gkae140. https://doi.org/10.1093/nar/gkae140

Wimberger, Sandra, et al. « Simultaneous Inhibition of DNA-PK and Polϴ Improves Integration Efficiency and Precision of Genome Editing ». Nature Communications, [...]

Sighinolfi, Silvia, et al. « Intracellular Iron Controls HSC Metabolism By Affecting Mitochondrial Fitness ». Blood, vol. 142, no Supplement 1, novembre [...]

P, Zarghamian, et al. « Clinical Genome Editing to Treat Sickle Cell Disease-A Brief Update ». Frontiers in Medicine, vol. 9, janvier [...]

Antoniou, Panagiotis, et al. « Base-Editing-Mediated Dissection of a γ-Globin Cis-Regulatory Element for the Therapeutic Reactivation of Fetal Hemoglobin Expression ». Nature Communications, [...]