Detalhe da pesquisa
1.
ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
Mol Ther
; 29(11): 3230-3242, 2021 11 03.
Artigo
Inglês
| MEDLINE | ID: mdl-33775910
2.
Epigenetic control of multiple genes with a lentiviral vector encoding transcriptional repressors fused to compact zinc finger arrays.
Mol Ther Methods Clin Dev
; 32(2): 101255, 2024 Jun 13.
Artigo
Inglês
| MEDLINE | ID: mdl-38715734
3.
Magselectofection: an integrated method of nanomagnetic separation and genetic modification of target cells.
Blood
; 117(16): e171-81, 2011 Apr 21.
Artigo
Inglês
| MEDLINE | ID: mdl-21357765
4.
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
5.
Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.
Mol Ther Methods Clin Dev
; 17: 1014-1025, 2020 Jun 12.
Artigo
Inglês
| MEDLINE | ID: mdl-32462050
6.
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Mol Ther Methods Clin Dev
; 18: 607-619, 2020 Sep 11.
Artigo
Inglês
| MEDLINE | ID: mdl-32775495
7.
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE.
Mol Ther Methods Clin Dev
; 8: 152-165, 2018 Mar 16.
Artigo
Inglês
| MEDLINE | ID: mdl-29687034
8.
Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice.
Hum Gene Ther
; 25(10): 905-14, 2014 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-25222508
9.
Comprehensive investigation of parameter choice in viral integration site analysis and its effects on the gene annotations produced.
Hum Gene Ther
; 23(11): 1209-19, 2012 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-22909036