Detalhe da pesquisa
1.
Gene therapy for inborn errors of immunity: Base editing comes into play.
Cell
; 186(7): 1302-1304, 2023 03 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-37001495
2.
Chromothriptic cure of WHIM syndrome.
Cell
; 160(4): 686-699, 2015 Feb 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-25662009
3.
Lentiviral Gene Therapy for Artemis-Deficient SCID.
N Engl J Med
; 387(25): 2344-2355, 2022 12 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-36546626
4.
CRISPR/Cas9-mediated Cxcr4 disease allele inactivation for gene therapy in a mouse model of WHIM syndrome.
Blood
; 142(1): 23-32, 2023 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36928087
5.
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Blood
; 141(9): 1007-1022, 2023 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36332160
6.
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.
J Allergy Clin Immunol
; 153(1): 287-296, 2024 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37793572
7.
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study.
J Allergy Clin Immunol
; 2024 Jan 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-38290608
8.
Homozygous IL37 mutation associated with infantile inflammatory bowel disease.
Proc Natl Acad Sci U S A
; 118(10)2021 03 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33674380
9.
Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers.
J Clin Immunol
; 43(8): 1964-1973, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37620741
10.
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
11.
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
12.
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
13.
Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease.
BMC Gastroenterol
; 23(1): 166, 2023 May 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-37208598
14.
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
15.
Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy.
J Clin Immunol
; 42(5): 1026-1035, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35445907
16.
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
17.
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
18.
Febrile neutropenia management and outcomes in hematopoietic cell transplantation for chronic granulomatous disease.
Transpl Infect Dis
; 24(2): e13815, 2022 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-35191140
19.
Gastrointestinal Computed Tomography Findings in Chronic Granulomatous Disease with Subgroup Clinicopathologic Analysis.
Dig Dis Sci
; 67(5): 1831-1842, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33934254
20.
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