Detalhe da pesquisa
1.
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Hum Mol Genet
; 33(3): 211-223, 2024 Jan 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-37819629
2.
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia.
Am J Hum Genet
; 110(3): 531-547, 2023 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36809767
3.
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
Hum Mol Genet
; 31(1): 82-96, 2021 12 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-34368854
4.
Quantitation of Pax-6 protein in ocular impression cytology samples using an electrochemiluminescence immunoassay.
Anal Biochem
; 656: 114876, 2022 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36058293
5.
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Hum Mol Genet
; 25(10): 1885-1899, 2016 05 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26931466
6.
PTC124 targets genetic disorders caused by nonsense mutations.
Nature
; 447(7140): 87-91, 2007 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-17450125
7.
Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia.
Front Oncol
; 12: 832816, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35223511
8.
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent.
Mol Cancer Ther
; 20(10): 1846-1857, 2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34315764
9.
Chemical modifications of G418 (geneticin): Synthesis of novel readthrough aminoglycosides results in an improved in vitro safety window but no improvements in vivo.
Carbohydr Res
; 495: 108058, 2020 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-32658832
10.
Effective Delivery of a Microtubule Polymerization Inhibitor Synergizes with Standard Regimens in Models of Pancreatic Ductal Adenocarcinoma.
Clin Cancer Res
; 25(18): 5548-5560, 2019 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-31175095
11.
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
PLoS One
; 13(10): e0206158, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-30359426
12.
Structure-activity relationship (SAR) optimization of 6-(indol-2-yl)pyridine-3-sulfonamides: identification of potent, selective, and orally bioavailable small molecules targeting hepatitis C (HCV) NS4B.
J Med Chem
; 57(5): 2121-35, 2014 Mar 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-24266880