Detalhe da pesquisa
1.
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
EMBO J
; 42(23): e114188, 2023 Dec 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37916874
2.
Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.
Semin Immunol
; 66: 101731, 2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36863140
3.
Transplantation after CD45-ADC corrects Rag1 immunodeficiency in congenic and haploidentical settings.
J Allergy Clin Immunol
; 153(1): 341-348.e3, 2024 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37567393
4.
RAG gene defects at the verge of immunodeficiency and immune dysregulation.
Immunol Rev
; 287(1): 73-90, 2019 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30565244
5.
Immune dysregulation in patients with RAG deficiency and other forms of combined immune deficiency.
Blood
; 135(9): 610-619, 2020 02 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-31942628
6.
Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency.
J Allergy Clin Immunol
; 147(1): 309-320.e6, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32387109
7.
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
em Inglês
| MEDLINE | ID: mdl-31949009
8.
Cutaneous barrier leakage and gut inflammation drive skin disease in Omenn syndrome.
J Allergy Clin Immunol
; 146(5): 1165-1179.e11, 2020 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32311393
9.
Insights into Cadmium-Induced Carcinogenesis through an In Vitro Study Using C3H10T1/2Cl8 Cells: The Multifaceted Role of Mitochondria.
Int J Mol Sci
; 22(19)2021 Oct 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34639177
10.
Gut Microbiota-Host Interactions in Inborn Errors of Immunity.
Int J Mol Sci
; 22(3)2021 Jan 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-33572538
11.
T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency.
Blood
; 132(22): 2362-2374, 2018 11 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-30254128
12.
Chromosome Transplantation: Correction of the Chronic Granulomatous Disease Defect in Mouse Induced Pluripotent Stem Cells.
Stem Cells
; 37(7): 876-887, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30895693
13.
Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.
J Allergy Clin Immunol
; 144(3): 825-838, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-30926529
14.
The microbiome and immunodeficiencies: Lessons from rare diseases.
J Autoimmun
; 98: 132-148, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30704941
15.
B lymphocytes limit senescence-driven fibrosis resolution and favor hepatocarcinogenesis in mouse liver injury.
Hepatology
; 67(5): 1970-1985, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29105104
16.
MicroRNA-127-3p controls murine hematopoietic stem cell maintenance by limiting differentiation.
Haematologica
; 104(9): 1744-1755, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-30792210
17.
Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation.
J Allergy Clin Immunol
; 142(3): 928-941.e8, 2018 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-29241731
18.
Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation.
J Allergy Clin Immunol
; 142(4): 1272-1284, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-29421274
19.
Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome.
J Allergy Clin Immunol
; 142(5): 1605-1617.e4, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29447842
20.
Murine Rankl-/- Mesenchymal Stromal Cells Display an Osteogenic Differentiation Defect Improved by a RANKL-Expressing Lentiviral Vector.
Stem Cells
; 35(5): 1365-1377, 2017 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28100034