Detalhe da pesquisa
1.
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy.
Am J Hum Genet
; 110(10): 1648-1660, 2023 10 05.
Artigo
Inglês
| MEDLINE | ID: mdl-37673065
2.
Nuclear architecture dictates HIV-1 integration site selection.
Nature
; 521(7551): 227-31, 2015 May 14.
Artigo
Inglês
| MEDLINE | ID: mdl-25731161
3.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human ß-globin locus.
Blood
; 131(17): 1960-1973, 2018 04 26.
Artigo
Inglês
| MEDLINE | ID: mdl-29519807
4.
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
Inglês
| MEDLINE | ID: mdl-30424953
5.
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
Blood
; 130(11): 1327-1335, 2017 09 14.
Artigo
Inglês
| MEDLINE | ID: mdl-28716862
6.
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.
Mol Ther
; 26(4): 1137-1153, 2018 04 04.
Artigo
Inglês
| MEDLINE | ID: mdl-29503198
7.
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.
Mol Ther
; 25(4): 839-854, 2017 04 05.
Artigo
Inglês
| MEDLINE | ID: mdl-28237839
8.
IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.
Blood
; 121(4): 573-84, 2013 Jan 24.
Artigo
Inglês
| MEDLINE | ID: mdl-23160470
9.
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
JAMA
; 313(15): 1550-63, 2015 Apr 21.
Artigo
Inglês
| MEDLINE | ID: mdl-25898053
10.
Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.
Mol Ther
; 21(9): 1695-704, 2013 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-23760447
11.
Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.
J Infect Dis
; 208(2): 235-43, 2013 Jul 15.
Artigo
Inglês
| MEDLINE | ID: mdl-23559464
12.
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.
Nat Med
; 12(12): 1397-402, 2006 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-17115047
13.
Gene therapies need new development models.
Nature
; 490(7418): 7, 2012 Oct 04.
Artigo
Inglês
| MEDLINE | ID: mdl-23038429
14.
Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.
Mol Ther
; 20(4): 798-807, 2012 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-22068429
15.
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Mol Ther Nucleic Acids
; 32: 229-246, 2023 Jun 13.
Artigo
Inglês
| MEDLINE | ID: mdl-37090420
16.
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Lancet Neurol
; 22(12): 1125-1139, 2023 12.
Artigo
Inglês
| MEDLINE | ID: mdl-37977713
17.
High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.
Blood
; 116(25): 5507-17, 2010 Dec 16.
Artigo
Inglês
| MEDLINE | ID: mdl-20864581
18.
C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.
J Cell Biol
; 177(6): 1037-49, 2007 Jun 18.
Artigo
Inglês
| MEDLINE | ID: mdl-17562792
19.
Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors.
PLoS Comput Biol
; 7(12): e1002292, 2011 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-22144885
20.
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Mol Ther
; 19(10): 1867-77, 2011 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-21750532