Biblio

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Journal Article

Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder DK, Cattoni A, Chernyshova L, et al. Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity. Blood. 2024.

Passerini L, Barzaghi F, Curto R, Sartirana C, Barera G, Tucci F, Albarello L, Mariani A, AlbertoTestoni P, Bazzigaluppi E, et al. Treatment with Rapamycin Can Restore Regulatory T Cell Function in IPEX Patients. J Allergy Clin Immunol. 2019.

Aiuti A, Naldini L. Safer conditioning for blood stem cell transplants. Nat Biotechnol. 2016;34(7):721-723.

Sauer AV, Di Lorenzo B, Carriglio N, Aiuti A. Progress in gene therapy for primary immunodeficiencies using lentiviral vectors. Curr Opin Allergy Clin Immunol. 2014.

Ferrua F, Cicalese MPia, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo MEster, Calbi V, Assanelli AAngelo, et al. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Lancet Haematol. 2019.

Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, et al. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet. 2016.

Marktel S, Scaramuzza S, Cicalese MPia, Giglio F, Galimberti S, Lidonnici MRosa, Calbi V, Assanelli A, Bernardo MEster, Rossi C, et al. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia. Nat Med. 2019.

Fratini ESophia, Migliavacca M, Barzaghi F, Fossati C, Giannelli S, Monti I, Casiraghi M, Ferrua F, Recupero S, Consiglieri G, et al. Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review. Front Immunol. 2023;14:1187959.

Hernández RJofra, Calabria A, Sanvito F, De Mattia F, Farinelli G, Scala S, Visigalli I, Carriglio N, De Simone M, Vezzoli M, et al. Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. Mol Ther. 2020.

Albert MH, Slatter MA, Gennery AR, Güngör T, Bakunina K, Markovitch B, Hazelaar S, Sirait T, Courteille V, Aiuti A, et al. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood. 2022.

Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, et al. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.

Scala S, Ferrua F, Basso-Ricci L, Dionisio F, Omrani M, Quaranta P, Hernández RJofra, Del Core L, Benedicenti F, Monti I, et al. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy. Nat Commun. 2023;14(1):3068.

Ferrua F, Marangoni F, Aiuti A, Roncarolo MGrazia. Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions. J Allergy Clin Immunol. 2020.

Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, et al. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024;16(745):eadi8214.

Colantuoni M, Hernández RJofra, Pettinato E, Basso-Ricci L, Magnani L, Andolfi G, Rigamonti C, Finardi A, Romeo V, Soldi M, et al. Constitutive IL-1RA production by modified immune cells protects against IL-1-mediated inflammatory disorders. Sci Transl Med. 2023;15(698):eade3856.

Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, H Gaspar B, Notarangelo LD, Grunebaum E. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2018.

Quaranta P, Basso-Ricci L, Hernández RJofra Jofr, Pacini G, Naldini MMaria, Barcella M, Seffin L, Pais G, Spinozzi G, Benedicenti F, et al. Circulating Hematopoietic Stem/Progenitor Cells subsets contribute to human hematopoietic homeostasis. Blood. 2024.

Consiglieri G, Ferrua F, Aiuti A, Cicalese MPia. A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation. J Clin Immunol. 2021.

Cesana D, Cicalese MPia, Calabria A, Merli P, Caruso R, Volpin M, Rudilosso L, Migliavacca M, Barzaghi F, Fossati C, et al. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID. Nat Commun. 2024;15(1):3662.

Fox T, Bueren J, Candotti F, Fischer A, Aiuti A, Lankester A, Booth C. Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nat Med. 2023.