Detalhe da pesquisa
1.
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Mol Ther
; 27(1): 178-187, 2019 01 02.
Artigo
Inglês
| MEDLINE | ID: mdl-30528089
2.
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
Inglês
| MEDLINE | ID: mdl-29580682
3.
Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies.
Hum Gene Ther
; 33(21-22): 1126-1141, 2022 11.
Artigo
Inglês
| MEDLINE | ID: mdl-35994386