Detalhe da pesquisa
1.
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction.
Gene Ther
; 30(3-4): 386-397, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36258038
2.
Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.
J Inherit Metab Dis
; 44(6): 1369-1381, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33896013
3.
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.
Nat Commun
; 13(1): 5012, 2022 08 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-36008405
4.
Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing.
PLoS One
; 16(1): e0245831, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-33493163
5.
The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.
Expert Opin Orphan Drugs
; 8(7): 245-256, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-33224636
6.
Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure.
Mol Ther Methods Clin Dev
; 18: 738-750, 2020 Sep 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32913881
7.
Ex Vivo Hepatocyte Reprograming Promotes Homology-Directed DNA Repair to Correct Metabolic Disease in Mice After Transplantation.
Hepatol Commun
; 3(4): 558-573, 2019 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-30976745
8.
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine.
J Vis Exp
; (141)2018 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30451238