Detalhe da pesquisa
1.
Assessment of PABPN1 nuclear inclusions on a large cohort of patients and in a human xenograft model of oculopharyngeal muscular dystrophy.
Acta Neuropathol
; 144(6): 1157-1170, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36197469
2.
Trehalose alleviates the phenotype of Machado-Joseph disease mouse models.
J Transl Med
; 18(1): 161, 2020 04 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32272938
3.
TAT-MTS-MCM fusion proteins reduce MMA levels and improve mitochondrial activity and liver function in MCM-deficient cells.
J Cell Mol Med
; 22(3): 1601-1613, 2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29265583
4.
Frataxin-deficient neurons and mice models of Friedreich ataxia are improved by TAT-MTScs-FXN treatment.
J Cell Mol Med
; 22(2): 834-848, 2018 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28980774
5.
Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy.
J Neuromuscul Dis
; 4(3): 237-249, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-28598854
6.
Heterologous mitochondrial targeting sequences can deliver functional proteins into mitochondria.
Int J Biochem Cell Biol
; 81(Pt A): 48-56, 2016 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-27771440
7.
A flow cytometry-based reporter assay identifies macrolide antibiotics as nonsense mutation read-through agents.
J Mol Med (Berl)
; 94(4): 469-82, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26620677
8.
Erratum to: A flow cytometry-based reporter assay identifies macrolide antibiotics as nonsense mutation read-through agents.
J Mol Med (Berl)
; 94(4): 483, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26897011