Detalhe da pesquisa
1.
Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress.
Nat Immunol
; 23(10): 1470-1483, 2022 10.
Artigo
Inglês
| MEDLINE | ID: mdl-36138183
2.
Gene therapy using haematopoietic stem and progenitor cells.
Nat Rev Genet
; 22(4): 216-234, 2021 04.
Artigo
Inglês
| MEDLINE | ID: mdl-33303992
3.
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.
Blood
; 143(19): 1937-1952, 2024 May 09.
Artigo
Inglês
| MEDLINE | ID: mdl-38446574
4.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
Inglês
| MEDLINE | ID: mdl-34788506
5.
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis.
Blood
; 139(13): 2066-2079, 2022 03 31.
Artigo
Inglês
| MEDLINE | ID: mdl-35100336
6.
Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study.
Blood
; 140(14): 1635-1649, 2022 10 06.
Artigo
Inglês
| MEDLINE | ID: mdl-35344580
7.
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs.
Mol Ther
; 31(1): 230-248, 2023 01 04.
Artigo
Inglês
| MEDLINE | ID: mdl-35982622
8.
Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity.
J Allergy Clin Immunol
; 152(4): 984-996.e10, 2023 10.
Artigo
Inglês
| MEDLINE | ID: mdl-37390899
9.
Genetic determinants of type 1 diabetes in individuals with weak evidence of islet autoimmunity at disease onset.
Diabetologia
; 66(4): 695-708, 2023 04.
Artigo
Inglês
| MEDLINE | ID: mdl-36692510
10.
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
Inglês
| MEDLINE | ID: mdl-35065785
11.
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
Inglês
| MEDLINE | ID: mdl-35945378
12.
The Impact of SARS-CoV-2 Infection in Patients with Inborn Errors of Immunity: the Experience of the Italian Primary Immunodeficiencies Network (IPINet).
J Clin Immunol
; 42(5): 935-946, 2022 07.
Artigo
Inglês
| MEDLINE | ID: mdl-35445287
13.
CXCR5-CXCL13 axis markers in full-term and preterm human neonates in the first weeks of life.
Eur J Immunol
; 51(5): 1289-1292, 2021 05.
Artigo
Inglês
| MEDLINE | ID: mdl-33491181
14.
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
Inglês
| MEDLINE | ID: mdl-34547335
15.
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases.
Mol Ther
; 29(2): 489-504, 2021 02 03.
Artigo
Inglês
| MEDLINE | ID: mdl-33221437
16.
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.
Artigo
Inglês
| MEDLINE | ID: mdl-33010230
17.
Coronavirus disease 2019 in patients with inborn errors of immunity: An international study.
J Allergy Clin Immunol
; 147(2): 520-531, 2021 Feb.
Artigo
Inglês
| MEDLINE | ID: mdl-32980424
18.
Expanded circulating hematopoietic stem/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis.
Haematologica
; 106(1): 74-86, 2021 01 01.
Artigo
Inglês
| MEDLINE | ID: mdl-31949009
19.
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
Inglês
| MEDLINE | ID: mdl-32817285
20.
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
J Inherit Metab Dis
; 44(5): 1151-1164, 2021 09.
Artigo
Inglês
| MEDLINE | ID: mdl-33855715