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
em Inglês
| MEDLINE
| ID: mdl-33775910
2.
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Mol Ther;
27(1): 178-187, 2019 01 02.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30528089
3.
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
Mol Ther;
26(4): 1127-1136, 2018 04 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29580682
4.
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
Blood;
126(15): 1777-84, 2015 Oct 08.
Artigo
em Inglês
| MEDLINE
| ID: mdl-26297739
5.
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
em Inglês
| MEDLINE
| ID: mdl-32775495