Detalhe da pesquisa
1.
Biallelic human SHARPIN loss of function induces autoinflammation and immunodeficiency.
Nat Immunol
; 25(5): 764-777, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38609546
2.
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
3.
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
4.
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
5.
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
6.
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
7.
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
8.
The promise of in vivo HSC prime editing.
Blood
; 141(17): 2039-2040, 2023 04 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37103948
9.
Myeloid Conditioning with c-kit-Targeted CAR-T Cells Enables Donor Stem Cell Engraftment.
Mol Ther
; 26(5): 1181-1197, 2018 05 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29622475
10.
Cytoskeletal abnormalities and neutrophil dysfunction in WDR1 deficiency.
Blood
; 128(17): 2135-2143, 2016 10 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-27557945
11.
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
12.
Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers.
Mol Ther
; 25(1): 44-53, 2017 01 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28129126
13.
Molecular Analysis of Neutrophil Differentiation from Human Induced Pluripotent Stem Cells Delineates the Kinetics of Key Regulators of Hematopoiesis.
Stem Cells
; 34(6): 1513-26, 2016 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-26866427
14.
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
15.
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
16.
Enhancing implementation of the I-PASS Handoff Tool Using a Provider Handoff Task Force at a Comprehensive Cancer Center.
Jt Comm J Qual Patient Saf
; 2024 Mar 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-38584053
17.
Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting.
Blood
; 117(21): 5561-72, 2011 May 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-21411759
18.
Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells.
Cell Rep Methods
; 3(4): 100460, 2023 04 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-37159663
19.
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
Nat Commun
; 13(1): 3710, 2022 06 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-35764638
20.
Using CRISPR/Cas9 for Gene Knockout in Immunodeficient NSG Mice.
Methods Mol Biol
; 1874: 139-168, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30353512