Detalhe da pesquisa
1.
Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.
Cell
; 186(7): 1398-1416.e23, 2023 03 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-36944331
2.
Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair.
Stem Cells
; 37(2): 284-294, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30372555
3.
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
4.
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
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.
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.
Cytotherapy
; 19(9): 1096-1112, 2017 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28733131
7.
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
8.
Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy.
Stem Cells
; 33(5): 1532-42, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25588820
9.
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
iScience
; 25(6): 104374, 2022 Jun 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-35633935
10.
Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells.
Front Genome Ed
; 2: 601541, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-34713224
11.
Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies.
Sci Adv
; 6(43)2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33097539
12.
Promise of gene therapy to treat sickle cell disease.
Expert Opin Biol Ther
; 18(11): 1123-1136, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30324810
13.
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
14.
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
15.
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
16.
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.
Exp Hematol
; 43(5): 346-351, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25681747
17.
ß-globin gene transfer to human bone marrow for sickle cell disease.
J Clin Invest
; 2013 Jul 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-23863630
18.
Safer vectors for gene therapy of primary immunodeficiencies.
Curr Gene Ther
; 9(4): 291-305, 2009 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-19534652