Detalhe da pesquisa
1.
Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts.
iScience
; 27(4): 109530, 2024 Apr 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-38577102
2.
Efficient and Fast Generation of Relevant Disease Mouse Models by In Vitro and In Vivo Gene Editing of Zygotes.
CRISPR J
; 5(3): 422-434, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35686982
3.
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Mol Ther Methods Clin Dev
; 22: 350-359, 2021 Sep 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34514027