Biblio
Targeting autophagy potentiates tyrosine kinase inhibitor-induced cell death in Philadelphia chromosome-positive cells, including primary CML stem cells. J Clin Invest. 2013;123(8):3634.
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells. Mol Ther. 2023.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia. Nat Med. 2019.
Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche. Blood. 2020.
Gene therapy for Hemoglobinopathies. Hum Gene Ther. 2023.
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