Giuliana Ferrari

Vita-Salute San Raffaele University — Italy

Medicine · Health Sciences

51h-index13.5kcitations237works4.02yr avg
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Research Topics

Hemoglobinopathies and Related Disorders(52), Virus-based gene therapy research(52), CRISPR and Genetic Engineering(43), Muscle Physiology and Disorders(23), Erythrocyte Function and Pathophysiology(21)

Publications237 total

Base editing of β <sup>0</sup> -thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies
Science Translational Medicine·2025
Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche
Cell Reports Medicine·2025· 1 citedOpen Access
Decoding the role of inflammatory signaling in shaping HSC heterogeneity and engraftment in gene therapy-treated β-thalassemia pediatric patients
Blood·2025
Base editing of β <sup>0</sup> thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies
2025Open Access
Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia
Nature Communications·2025· 1 citedOpen Access
Immunomodulatory effects of dental pulp stem cells on lymphocytes and monocytes from patients with rheumatoid arthritis
Clinical and Experimental Rheumatology·2025· 1 citedOpen Access
Rational Modification of a Cross-Linker for Improved Flexible Protein Structure Modeling
Analytical Chemistry·2025· 4 citedOpen Access
Long-term lineage commitment in haematopoietic stem cell gene therapy
Nature·2024· 37 citedOpen Access
FATORES DE RISCO PARA DEFICIÊNCIA DE VITAMINA D EM PACIENTES PEDIÁTRICOS COM DOENÇA FALCIFORME
Hematology Transfusion and Cell Therapy·2024Open Access
3117 – HSCS IN Β-THALASSEMIA HAVE A REDUCED DORMANCY AND AN ERYTHROID BIASED LINEAGE COMMITMENT IN RESPONSE TO ALTERED TGFΒ SIGNALING IN THE BM MICROENVIRONMENT
Experimental Hematology·2024
3050 – UNIQUE TNFΑ-NFKB SIGNALING DYNAMICS REGULATES HEMATOPOIETIC STEM CELL LINEAGE FATE IN BETA-THALASSEMIA
Experimental Hematology·2024
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells
Molecular Therapy·2023· 12 citedOpen Access
Intracellular Iron Controls HSC Metabolism By Affecting Mitochondrial Fitness
Blood·2023· 2 cited
FGF23 Inhibition Improves Hematopoietic Stem Cell Transplantation in β-Thalassemia
Blood·2023Open Access
Gene Therapy for Hemoglobinopathies
Human Gene Therapy·2023· 10 cited
Table of Contents
Kidney International·2023Open Access
Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia
Science Translational Medicine·2023· 12 cited
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
Cell stem cell·2023· 61 citedOpen Access
A single approach to targeting transferrin receptor 2 corrects iron and erythropoietic defects in murine models of anemia of inflammation and chronic kidney disease
Kidney International·2023· 18 cited
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
Molecular Therapy — Nucleic Acids·2023· 14 citedOpen Access
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Frequent Co-authors

Fulvio Mavilio(23), Alessandro Aiuti(9), Annarita Miccio(7), Maria Rosa Lidonnici(7), Giulio Cossu(6), Claudio Bordignon(6), Claudia Rossi(6), Chiara Bonini(4), Francesco Saverio Tedesco(4), Sarah Marktel(4), Luigi Naldini(4), Francesco Lotti(4), Laura Silvestri(4), Giovanni Salvatori(4), Paolo Servida(3), Maurilio Ponzoni(3), Silvano Rossini(3), Catia Traversari(3), Nadia Nobili(3), Daniela Maggioni(3)