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Cell Stem Cell|February 7, 2025
Two is better than one: Advancing gene therapy protocols for enhanced safety and efficacyGiulia Hardouin, Annarita MiccioTrends in Molecular Medicine|January 10, 2025
Gene therapy for β-thalassemia: current and future optionsGiulia Hardouin, Annarita Miccio, Megane BrussonAnnual Review of Genomics and Human Genetics|August 25, 2023
Sickle Cell Disease: From Genetics to Curative ApproachesGiulia Hardouin, Elisa Magrin, Alice Corsia, et al.Blood|December 12, 2022
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutationGiulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 24, 2024
Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responsesGiacomo Frati, Megane Brusson, Gilles Sartre, et al.Cell Reports. Medicine|September 27, 2025
Multiplex base editing of BCL11A regulatory elements to treat sickle cell diseaseLetizia Fontana, Pierre Martinucci, Simone Amistadi, et al.Seminars in Arthritis and Rheumatism|March 31, 2020
Severe axial and pelvifemoral muscle damage in immune-mediated necrotizing myopathy evaluated by whole-body MRIOcéane Landon-Cardinal, Cédi Koumako, Giulia Hardouin, et al.Science Advances|September 12, 2020
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotypeLeslie Weber, Giacomo Frati, Tristan Felix, et al.Science Translational Medicine|November 26, 2025
Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathiesGiulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, et al.Nature Communications|November 5, 2022
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expressionPanagiotis Antoniou, Giulia Hardouin, Pierre Martinucci, et al.Pageof 2