Detalhe da pesquisa
1.
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
2.
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
3.
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