Detalhe da pesquisa
1.
Post-Translational Modifications of the DUX4 Protein Impact Toxic Function in FSHD Cell Models.
Ann Neurol
; 94(2): 398-413, 2023 08.
Artigo
Inglês
| MEDLINE | ID: mdl-37186119
2.
RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro.
RNA
; 25(9): 1211-1217, 2019 09.
Artigo
Inglês
| MEDLINE | ID: mdl-31209064
3.
RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.
Mol Ther
; 28(7): 1706-1716, 2020 07 08.
Artigo
Inglês
| MEDLINE | ID: mdl-32353324
4.
Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4.
Hum Mol Genet
; 25(20): 4577-4589, 2016 10 15.
Artigo
Inglês
| MEDLINE | ID: mdl-28173143
5.
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy.
Mol Ther
; 20(7): 1417-23, 2012 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-22508491
6.
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis.
bioRxiv
; 2023 Apr 18.
Artigo
Inglês
| MEDLINE | ID: mdl-37131694
7.
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis.
Sci Adv
; 9(27): eadd9984, 2023 07 07.
Artigo
Inglês
| MEDLINE | ID: mdl-37418531
8.
DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo.
Ann Neurol
; 69(3): 540-52, 2011 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-21446026
9.
RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1).
Mol Ther
; 19(11): 2048-54, 2011 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-21730972
10.
The DUX4 protein is a co-repressor of the progesterone and glucocorticoid nuclear receptors.
FEBS Lett
; 596(20): 2644-2658, 2022 10.
Artigo
Inglês
| MEDLINE | ID: mdl-35662006
11.
Meeting report: the 2021 FSHD International Research Congress.
Skelet Muscle
; 12(1): 1, 2022 01 17.
Artigo
Inglês
| MEDLINE | ID: mdl-35039091
12.
A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice.
J Clin Invest
; 132(13)2022 07 01.
Artigo
Inglês
| MEDLINE | ID: mdl-35579942
13.
PP2A:B56{epsilon}, a substrate of caspase-3, regulates p53-dependent and p53-independent apoptosis during development.
J Biol Chem
; 285(45): 34493-502, 2010 Nov 05.
Artigo
Inglês
| MEDLINE | ID: mdl-20807766
14.
Construction of permanently inducible miRNA-based expression vectors using site-specific recombinases.
BMC Biotechnol
; 11: 107, 2011 Nov 16.
Artigo
Inglês
| MEDLINE | ID: mdl-22087765
15.
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.
Nat Med
; 10(8): 816-20, 2004 Aug.
Artigo
Inglês
| MEDLINE | ID: mdl-15235598
16.
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
Nat Med
; 8(3): 253-61, 2002 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-11875496
17.
Molecular dissection of dystrophin identifies the docking site for nNOS.
Proc Natl Acad Sci U S A
; 110(2): 387-8, 2013 Jan 08.
Artigo
Inglês
| MEDLINE | ID: mdl-23277550
18.
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.
Proc Natl Acad Sci U S A
; 105(15): 5868-73, 2008 Apr 15.
Artigo
Inglês
| MEDLINE | ID: mdl-18398004
19.
Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes.
Mol Ther Nucleic Acids
; 23: 476-486, 2021 Mar 05.
Artigo
Inglês
| MEDLINE | ID: mdl-33510937
20.
Is Upregulation of Sarcolipin Beneficial or Detrimental to Muscle Function?
Front Physiol
; 12: 633058, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-33732165