Detalhe da pesquisa
1.
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
2.
Targeting strategies for modulating pre-mRNA splicing with small molecules: Recent advances.
Drug Discov Today
; 28(1): 103431, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36356786
3.
A deep learning approach to identify gene targets of a therapeutic for human splicing disorders.
Nat Commun
; 12(1): 3332, 2021 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34099697
4.
Small molecule splicing modifiers with systemic HTT-lowering activity.
Nat Commun
; 12(1): 7299, 2021 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-34911927
5.
Discovery and Optimization of Indolyl-Containing 4-Hydroxy-2-Pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-negative Bacteria.
J Med Chem
; 61(10): 4456-4475, 2018 05 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-29727185
6.
Solution structure of a small protein containing a fluorinated side chain in the core.
Protein Sci
; 16(1): 14-9, 2007 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-17123960
7.
Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
J Med Chem
; 59(13): 6086-100, 2016 07 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-27299419
8.
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
J Med Chem
; 59(13): 6070-85, 2016 07 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-27299569
9.
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Science
; 345(6197): 688-93, 2014 Aug 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-25104390
10.
Correction to Discovery and Optimization of Indolyl-Containing 4-Hydroxy-2-pyridone Type II DNA Topoisomerase Inhibitors Active against Multidrug Resistant Gram-Negative Bacteria.
J Med Chem
; 61(20): 9394, 2018 Oct 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-30351051
11.
Solution structure of a small protein containing a fluorinated side chain in the core.
Protein Sci
; 16(9): 2089, 2007 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-17915350
12.
Asymmetric cooperative catalysis of strong Brønsted acid-promoted reactions using chiral ureas.
Science
; 327(5968): 986-90, 2010 Feb 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-20167783
13.
Stabilizing and destabilizing effects of phenylalanine --> F5-phenylalanine mutations on the folding of a small protein.
J Am Chem Soc
; 128(50): 15932-3, 2006 Dec 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-17165695
14.
Backbone thioester exchange: a new approach to evaluating higher order structural stability in polypeptides.
J Am Chem Soc
; 126(36): 11172-4, 2004 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-15355097
15.
Stereoselective synthesis of 3-substituted 2-aminocyclopentanecarboxylic acid derivatives and their incorporation into short 12-helical beta-peptides that fold in water.
J Am Chem Soc
; 124(42): 12447-52, 2002 Oct 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-12381185