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
Inglês
| MEDLINE | ID: mdl-36944331
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.
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
4.
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
5.
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
6.
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
7.
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
8.
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
9.
The chromodomains of CHD1 are critical for enzymatic activity but less important for chromatin localization.
Nucleic Acids Res
; 39(8): 3103-15, 2011 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-21177652
10.
Apheresis of Deceased Donors as a New Source of Mobilized Peripheral Blood Hematopoietic Stem Cells for Transplant Tolerance.
Transplantation
; 107(2): 504-510, 2023 02 01.
Artigo
Inglês
| MEDLINE | ID: mdl-35974436
11.
Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants.
Blood Adv
; 6(21): 5732-5736, 2022 11 08.
Artigo
Inglês
| MEDLINE | ID: mdl-35914227
12.
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
iScience
; 25(6): 104374, 2022 Jun 17.
Artigo
Inglês
| MEDLINE | ID: mdl-35633935
13.
PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a ß-Globin Lentiviral Vector.
Mol Ther Methods Clin Dev
; 13: 390-398, 2019 Jun 14.
Artigo
Inglês
| MEDLINE | ID: mdl-31024981
14.
IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System.
Clin Cancer Res
; 25(3): 1000-1011, 2019 02 01.
Artigo
Inglês
| MEDLINE | ID: mdl-30409823
15.
Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer.
Cell Stem Cell
; 25(4): 542-557.e9, 2019 10 03.
Artigo
Inglês
| MEDLINE | ID: mdl-31495780
16.
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
Hum Gene Ther
; 29(10): 1153-1166, 2018 10.
Artigo
Inglês
| MEDLINE | ID: mdl-30198339
17.
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling ßAS3 Globin for Gene Therapy for Sickle Cell Disease.
Mol Ther Methods Clin Dev
; 11: 167-179, 2018 Dec 14.
Artigo
Inglês
| MEDLINE | ID: mdl-30533448
18.
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
Inglês
| MEDLINE | ID: mdl-29847792
19.
Purification and characterisation of the E7 oncoproteins of the high-risk human papillomavirus types 16 and 18.
J Virol Methods
; 134(1-2): 30-5, 2006 Jun.
Artigo
Inglês
| MEDLINE | ID: mdl-16384614
20.
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
Inglês
| MEDLINE | ID: mdl-26029723