Biblio
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID. Nat Commun. 2024;15(1):3662.
Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism. Hemasphere. 2023;7(10):e953.
. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024;16(745):eadi8214.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review. Front Immunol. 2023;14:1187959.
Treatment with Rapamycin Can Restore Regulatory T Cell Function in IPEX Patients. J Allergy Clin Immunol. 2019.