Detalhe da pesquisa
1.
Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.
Mol Ther
; 27(8): 1389-1406, 2019 08 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-31178391
2.
Characterization of Gene Alterations following Editing of the ß-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.
Mol Ther
; 26(2): 468-479, 2018 02 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29221806
3.
A genome editing primer for the hematologist.
Blood
; 127(21): 2525-35, 2016 05 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-27053532
4.
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
Blood
; 127(7): 839-48, 2016 Feb 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-26758916
5.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Blood
; 125(17): 2597-604, 2015 Apr 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-25733580
6.
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
Mol Ther
; 24(9): 1561-9, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27406980
7.
Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model.
Nat Commun
; 14(1): 6291, 2023 10 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37828021
8.
Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.
Cell Rep
; 23(9): 2606-2616, 2018 05 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-29847792
9.
Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells.
Curr Protoc Stem Cell Biol
; 36: 5B.4.1-5B.4.10, 2016 Feb 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-26840227
10.
The human ankyrin 1 promoter insulator sustains gene expression in a ß-globin lentiviral vector in hematopoietic stem cells.
Mol Ther Methods Clin Dev
; 2: 15012, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26029723
11.
Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors.
Hum Gene Ther
; 25(7): 599-608, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24568341
12.
Analyzing CRISPR genome-editing experiments with CRISPResso.
Nat Biotechnol
; 34(7): 695-7, 2016 07 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-27404874