Giuliana Ferrari
Milan, Italy
Last Updated: 09/26/2026
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Found 38 publications
Claudio Bordignon.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: May 27, 2026
Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies.
Journal: Science translational medicine
Published: November 26, 2025
Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.
Journal: Cell reports. Medicine
Published: June 10, 2025
Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia.
Journal: Science translational medicine
Published: May 31, 2023
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: May 23, 2023
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy.
Journal: Cell stem cell
Published: February 01, 2023
Long-term lineage commitment in haematopoietic stem cell gene therapy.
Journal: Nature
Published: September 16, 2022
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Journal: Molecular therapy. Nucleic acids
Published: July 30, 2022
A single approach to targeting transferrin receptor 2 corrects iron and erythropoietic defects in murine models of anemia of inflammation and chronic kidney disease.
Journal: Kidney international
Published: July 27, 2022
Last Updated: 09/26/2026