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1.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artículo
en Inglés
| MEDLINE | ID: mdl-34788506
2.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artículo
en Inglés
| MEDLINE | ID: mdl-35065785
3.
A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.
J Clin Immunol
; 42(8): 1742-1747, 2022 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-35945378
4.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Mol Ther
; 29(1): 86-102, 2021 01 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-33010230
5.
Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.
Haematologica
; 106(2): 641-646, 2021 02 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-32817285
6.
Health-Related Quality of Life and Emotional Difficulties in Chronic Granulomatous Disease: Data on Adult and Pediatric Patients from Italian Network for Primary Immunodeficiency (IPINet).
J Clin Immunol
; 40(2): 289-298, 2020 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-31863244
7.
Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.
J Allergy Clin Immunol
; 144(3): 825-838, 2019 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-30926529
8.
Multiparametric Whole Blood Dissection: A one-shot comprehensive picture of the human hematopoietic system.
Cytometry A
; 91(10): 952-965, 2017 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-28609016
9.
Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response.
Mol Ther
; 24(10): 1873-1880, 2016 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-27456061
10.
Acute events in children with sickle cell disease in Italy during the COVID-19 pandemic: useful lessons learned.
Br J Haematol
; 194(5): 851-854, 2021 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-34036565
11.
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.
Mol Ther
; 22(8): 1472-1483, 2014 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-24869932
12.
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
Hepatology
; 68(6): 2434-2437, 2018 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-30014500
13.
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Sci Transl Med
; 16(745): eadi8214, 2024 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-38691622
14.
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Nat Commun
; 15(1): 3662, 2024 Apr 30.
Artículo
en Inglés
| MEDLINE | ID: mdl-38688902
15.
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Nat Med
; 30(2): 488-497, 2024 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-38355973
16.
Response to rituximab-based therapy and risk factor analysis in Epstein Barr Virus-related lymphoproliferative disorder after hematopoietic stem cell transplant in children and adults: a study from the Infectious Diseases Working Party of the European Group for Blood and Marrow Transplantation.
Clin Infect Dis
; 57(6): 794-802, 2013 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-23771985
17.
A prospective study on the efficacy of mobilization of autologous peripheral stem cells in pediatric oncohematology patients.
Transfusion
; 53(7): 1501-9, 2013 Jul.
Artículo
en Inglés
| MEDLINE | ID: mdl-23034006
18.
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.
Biomedicines
; 11(7)2023 Jun 24.
Artículo
en Inglés
| MEDLINE | ID: mdl-37509449
19.
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.
Front Immunol
; 14: 1187959, 2023.
Artículo
en Inglés
| MEDLINE | ID: mdl-37435083
20.
Pioglitazone as a novel therapeutic approach in chronic granulomatous disease.
J Allergy Clin Immunol
; 137(6): 1913-1915.e2, 2016 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-27056268