Detalhe da pesquisa
1.
Generating human artery and vein cells from pluripotent stem cells highlights the arterial tropism of Nipah and Hendra viruses.
Cell
; 185(14): 2523-2541.e30, 2022 07 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35738284
2.
Gene regulation in inborn errors of immunity: Implications for gene therapy design and efficacy.
Immunol Rev
; 322(1): 157-177, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38233996
3.
Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells.
Mol Ther
; 31(4): 1074-1087, 2023 Apr 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36793210
4.
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
5.
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Mol Ther
; 30(1): 223-237, 2022 01 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33794364
6.
CRISPR/Cas9 ß-globin gene targeting in human haematopoietic stem cells.
Nature
; 539(7629): 384-389, 2016 11 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-27820943
7.
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.
Mol Ther
; 29(6): 2008-2018, 2021 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33609734
8.
Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex.
Mol Ther
; 29(3): 1016-1027, 2021 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33678249
9.
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
10.
Gene Editing on Center Stage.
Trends Genet
; 34(8): 600-611, 2018 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-29908711
11.
Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.
J Clin Immunol
; 41(5): 852-862, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33650026
12.
Efficient scarless genome editing in human pluripotent stem cells.
Nat Methods
; 15(12): 1045-1047, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30504872
13.
Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αß T cell-depleted haploidentical hematopoietic stem cell transplantation.
Haematologica
; 106(3): 847-858, 2021 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32241852
14.
Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal Cells.
Mol Ther
; 28(9): 2007-2022, 2020 09 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-32531238
15.
Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Nucleic Acids Res
; 47(15): 7955-7972, 2019 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31147717
16.
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.
Blood
; 132(17): 1737-1749, 2018 10 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-30154114
17.
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Mol Ther
; 27(1): 137-150, 2019 01 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30424953
18.
A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell-Based Models for Cardiovascular Diseases.
Circ Res
; 120(10): 1561-1571, 2017 May 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-28246128
19.
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
Mol Ther
; 26(10): 2431-2442, 2018 10 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30005866
20.
Ethical and regulatory aspects of genome editing.
Blood
; 127(21): 2553-60, 2016 05 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-27053531