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.
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
5.
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
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
; 153(5): 1423-1431.e2, 2024 May.
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.
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
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.
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
12.
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
13.
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
14.
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
15.
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
16.
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
17.
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
18.
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
19.
Hematologically important mutations: X-linked chronic granulomatous disease (fourth update).
Blood Cells Mol Dis
; 90: 102587, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-34175765
20.
Hematologically important mutations: The autosomal forms of chronic granulomatous disease (third update).
Blood Cells Mol Dis
; 92: 102596, 2021 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-34547651