Biblio
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID. Nat Commun. 2024;15(1):3662.
CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study. J Allergy Clin Immunol. 2014.
Chronic Aichi virus infection as a cause of long-lasting multi-organ involvement in patients with primary immune deficiencies. Clin Infect Dis. 2023.
Exagamglogene Autotemcel for Severe Sickle Cell Disease. N Engl J Med. 2024.
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene. Hum Gene Ther. 2016;27(2):148-165.
Hematopoietic stem cell gene therapy: the point of view of the diseased bone marrow. Stem Cells Dev. 2016.
. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood. 2022.
A new step toward non-genotoxic conditioning prior to hematopoietic stem cell transplantation. Mol Ther. 2024.
. 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.
Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome. JAMA. 2015;313(15):1550-1563.
Therapeutic plasma exchange for life-threatening pediatric disorders. J Clin Apher. 2021.
Vascular access for optimal hematopoietic stem cell collection. J Clin Apher. 2020.