Biblio
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood. 2022.
Gene therapy for Hemoglobinopathies. Hum Gene Ther. 2023.
. Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche. Blood. 2020.
Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia. Sci Transl Med. 2023;15(698):eabq3679.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia. Nat Med. 2019.
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies. Mol Ther Nucleic Acids. 2023;32:229-246.