Detalhe da pesquisa
1.
Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients.
J Allergy Clin Immunol
; 151(6): 1634-1645, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36638922
2.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Blood
; 138(26): 2768-2780, 2021 12 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-34086870
3.
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.
Blood
; 137(19): 2598-2608, 2021 05 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-33623984
4.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Blood
; 138(15): 1304-1316, 2021 10 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-33974038
5.
Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function.
Blood
; 137(18): 2450-2462, 2021 05 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33512449
6.
Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease-associated inflammatory bowel disease.
J Gastroenterol Hepatol
; 38(12): 2083-2089, 2023 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-37743535
7.
Poor T-cell receptor ß repertoire diversity early posttransplant for severe combined immunodeficiency predicts failure of immune reconstitution.
J Allergy Clin Immunol
; 149(3): 1113-1119, 2022 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34384841
8.
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
N Engl J Med
; 380(16): 1525-1534, 2019 04 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-30995372
9.
Safety and Efficacy of Ustekinumab in the Inflammatory Bowel Disease of Chronic Granulomatous Disease.
Clin Gastroenterol Hepatol
; 20(2): 461-464.e2, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33813069
10.
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Gene Ther
; 28(6): 373-390, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33712802
11.
MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia disease.
Cytotherapy
; 23(3): 203-210, 2021 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33051095
12.
Progressive B Cell Loss in Revertant X-SCID.
J Clin Immunol
; 40(7): 1001-1009, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32681206
13.
The promise of in vivo HSC prime editing.
Blood
; 141(17): 2039-2040, 2023 04 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37103948
14.
Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction.
Mol Ther
; 25(2): 321-330, 2017 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28153086
15.
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Mol Ther
; 23(1): 147-57, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25288370
16.
Enhancers are major targets for murine leukemia virus vector integration.
J Virol
; 88(8): 4504-13, 2014 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-24501411
17.
Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34+ cells.
Blood
; 121(14): e98-107, 2013 Apr 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-23386128
18.
Mobilization characteristics and strategies to improve hematopoietic progenitor cell mobilization and collection in patients with chronic granulomatous disease and severe combined immunodeficiency.
Transfusion
; 55(2): 265-74, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25143186
19.
CXCR4/IgG-expressing plasma cells are associated with human gastrointestinal tissue inflammation.
J Allergy Clin Immunol
; 133(6): 1676-85.e5, 2014 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-24373354
20.
Paravertebral mushroom: identification of a novel species of Phellinus as a human pathogen in chronic granulomatous disease.
J Clin Microbiol
; 52(7): 2726-9, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24829241