Annarita Miccio

Inserm — France

Biochemistry, Genetics and Molecular Biology · Life Sciences

34h-index3.8kcitations124works7.02yr avg
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Research Topics

CRISPR and Genetic Engineering(67), Hemoglobinopathies and Related Disorders(58), Virus-based gene therapy research(28), Prenatal Screening and Diagnostics(16), Iron Metabolism and Disorders(15)

Publications124 total

Base editing of β <sup>0</sup> -thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies
Science Translational Medicine·2025
Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
Cell Reports Medicine·2025Open Access
VEXAS anemia is a mosaic erythroblastopenia
Blood·2025· 1 citedOpen Access
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
Blood·2025· 3 citedOpen Access
Base editing of β <sup>0</sup> thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies
2025Open Access
Advancing the Integration of ‘Basic/Fundamental’ and Translational Cellular and Gene Therapy Science within the EBMT: Accelerating the Pathway to Progress
Bone Marrow Transplantation·2025· 1 cited
Dissecting the epigenetic regulation of the fetal hemoglobin genes to unravel a novel therapeutic approach for β-hemoglobinopathies
Nucleic Acids Research·2025· 1 citedOpen Access
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Nature Communications·2025· 10 citedOpen Access
Two is better than one: Advancing gene therapy protocols for enhanced safety and efficacy
Cell stem cell·2025
Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group
Molecular Therapy — Nucleic Acids·2025· 30 citedOpen Access
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
2025· 3 citedOpen Access
Gene therapy for β-thalassemia: current and future options
Trends in Molecular Medicine·2025· 11 cited
Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies
médecine/sciences·2025Open Access
Multiplex base editing of <i>BCL11A</i> regulatory elements to treat sickle cell disease
2024· 1 citedOpen Access
VEXAS anemia is a mosaic erythroblastopenia
2024· 2 citedOpen Access
Vexas Syndrome Is a New Cause of p53-Mediated Erythroblastopenia
Blood·2024· 7 citedOpen Access
Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
Nature Communications·2024· 10 citedOpen Access
Cyclosporin H Improves the Transduction of CD34 <sup>+</sup> Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease
Human Gene Therapy·2024· 1 citedOpen Access
Treating Sickle Cell Disease: Gene Therapy Approaches
The Annual Review of Pharmacology and Toxicology·2024· 10 citedOpen Access
Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses
Molecular Therapy·2024· 19 citedOpen Access
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Frequent Co-authors

Marina Cavazzana(16), Fulvio Mavilio(15), Giuliana Ferrari(11), Tristan Félix(10), Elisa Magrin(10), Oriana Romano(9), Mario Amendola(8), Wassim El Nemer(7), Anne Chalumeau(7), Chiara Antoniani(6), Giacomo Frati(6), Laure Joseph(6), Panagiotis Antoniou(6), Mégane Brusson(6), Isabelle André‐Schmutz(5), Vasco Meneghini(5), Ermanno Rizzi(5), Marco Severgnini(5), Giulia Hardouin(5), Jean‐Paul Concordet(5)