Detalhe da pesquisa
1.
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Blood
; 141(9): 1007-1022, 2023 03 02.
Artigo
Inglês
| MEDLINE | ID: mdl-36332160
2.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
N Engl J Med
; 384(21): 2002-2013, 2021 05 27.
Artigo
Inglês
| MEDLINE | ID: mdl-33974366
3.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Blood
; 138(15): 1304-1316, 2021 10 14.
Artigo
Inglês
| MEDLINE | ID: mdl-33974038
4.
Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small ß-Globin Locus Control Region Elements.
Mol Ther
; 28(1): 328-340, 2020 01 08.
Artigo
Inglês
| MEDLINE | ID: mdl-31628051
5.
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
Inglês
| MEDLINE | ID: mdl-30372555
6.
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
Inglês
| MEDLINE | ID: mdl-31178391
7.
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
Inglês
| MEDLINE | ID: mdl-29221806
8.
Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells.
Mol Ther
; 25(9): 2163-2175, 2017 09 06.
Artigo
Inglês
| MEDLINE | ID: mdl-28663101
9.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Blood
; 125(17): 2597-604, 2015 Apr 23.
Artigo
Inglês
| MEDLINE | ID: mdl-25733580
10.
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
Inglês
| MEDLINE | ID: mdl-28733131
11.
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
Mol Ther
; 24(9): 1561-9, 2016 09.
Artigo
Inglês
| MEDLINE | ID: mdl-27406980
12.
Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy.
Stem Cells
; 33(5): 1532-42, 2015 May.
Artigo
Inglês
| MEDLINE | ID: mdl-25588820
13.
Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling.
Stem Cells
; 32(6): 1503-14, 2014 Jun.
Artigo
Inglês
| MEDLINE | ID: mdl-24677652
14.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Mol Ther
; 22(3): 607-622, 2014 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-24256635
15.
Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus.
Mol Ther
; 21(9): 1705-17, 2013 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-23857176
16.
A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease.
Mol Ther Methods Clin Dev
; 32(2): 101254, 2024 Jun 13.
Artigo
Inglês
| MEDLINE | ID: mdl-38745893
17.
A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors.
J Transl Med
; 11: 23, 2013 Jan 29.
Artigo
Inglês
| MEDLINE | ID: mdl-23360526
18.
Envelope, please. And the award goes to .
Blood
; 124(8): 1203-4, 2014 Aug 21.
Artigo
Inglês
| MEDLINE | ID: mdl-25147372
19.
Improved lentiviral vector titers from a multi-gene knockout packaging line.
Mol Ther Oncolytics
; 23: 582-592, 2021 Dec 17.
Artigo
Inglês
| MEDLINE | ID: mdl-34938858
20.
ß-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production.
Stem Cell Reports
; 16(1): 198-211, 2021 01 12.
Artigo
Inglês
| MEDLINE | ID: mdl-33186538