Detalhe da pesquisa
1.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med;
385(21): 1929-1940, 2021 11 18.
Artigo
em Inglês
| 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.
Artigo
em Inglês
| 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.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35945378
4.
Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
J Pediatr;
240: 297-301.e5, 2022 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34547335
5.
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases.
Mol Ther;
29(2): 489-504, 2021 02 03.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33221437
6.
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.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32817285
7.
Treatment with rapamycin can restore regulatory T-cell function in IPEX patients.
J Allergy Clin Immunol;
145(4): 1262-1271.e13, 2020 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31874182
8.
Long-term results of high-dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: the Italian experience.
Br J Haematol;
170(3): 398-407, 2015 Aug.
Artigo
em Inglês
| MEDLINE
| ID: mdl-25891192
9.
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.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30014500
10.
A point-of-care test for facing the burden of undiagnosed celiac disease in the Mediterranean area: a pragmatic design study.
BMC Gastroenterol;
14: 219, 2014 Dec 18.
Artigo
em Inglês
| MEDLINE
| ID: mdl-25518884
11.
Celiac disease in the Mediterranean area.
BMC Gastroenterol;
14: 24, 2014 Feb 11.
Artigo
em Inglês
| MEDLINE
| ID: mdl-24517104
12.
Meta-analysis of genome-wide association studies in celiac disease and rheumatoid arthritis identifies fourteen non-HLA shared loci.
PLoS Genet;
7(2): e1002004, 2011 Feb.
Artigo
em Inglês
| MEDLINE
| ID: mdl-21383967
13.
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Sci Transl Med;
16(745): eadi8214, 2024 May.
Artigo
em Inglês
| 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.
Artigo
em Inglês
| 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.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38355973
16.
Early response does not predict outcome in children and adolescents with chronic myeloid leukaemia treated with high-dose imatinib.
Br J Haematol;
180(6): 895-898, 2018 03.
Artigo
em Inglês
| MEDLINE
| ID: mdl-27984633
17.
Diagnosis of Respiratory Syncytial Virus (RSV) infection in children by Respiratory Panel utilized during the COVID-19 pandemic.
Ann Ist Super Sanita;
59(1): 31-36, 2023.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36974702
18.
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.
Biomedicines;
11(7)2023 Jun 24.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37509449
19.
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.
Front Immunol;
14: 1187959, 2023.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37435083
20.
A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.
Nat Commun;
13(1): 1315, 2022 03 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35288539