Detalhe da pesquisa
1.
Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects.
PLoS Genet
; 16(11): e1009179, 2020 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33175853
2.
Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17.
Hum Mol Genet
; 27(17): 2978-2985, 2018 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29790927
3.
Progress and prospects of gene therapy clinical trials for the muscular dystrophies.
Hum Mol Genet
; 25(R1): R9-17, 2016 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26450518
4.
AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility.
Mol Ther
; 24(2): 240-250, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26388461
5.
Muscle structure influences utrophin expression in mdx mice.
PLoS Genet
; 10(6): e1004431, 2014 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-24922526
6.
Validation of ultrasonography for non-invasive assessment of diaphragm function in muscular dystrophy.
J Physiol
; 594(24): 7215-7227, 2016 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27570057
7.
Recombinant adeno-associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia.
J Neurochem
; 136 Suppl 1: 49-62, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25708596
8.
Phosphorylation within the cysteine-rich region of dystrophin enhances its association with ß-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.
Hum Mol Genet
; 23(25): 6697-711, 2014 Dec 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-25082828
9.
Dystrobrevin increases dystrophin's binding to the dystrophin-glycoprotein complex and provides protection during cardiac stress.
J Mol Cell Cardiol
; 76: 106-15, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25158611
10.
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle.
Res Sq
; 2023 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37398370
11.
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle.
Skelet Muscle
; 13(1): 16, 2023 09 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37705069
12.
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model.
Mol Ther Methods Clin Dev
; 28: 344-354, 2023 Mar 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36874243
13.
Cell based dATP delivery as a therapy for chronic heart failure.
bioRxiv
; 2023 Apr 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37162854
14.
Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.
Mol Ther
; 19(1): 36-45, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-20859263
15.
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model.
JCI Insight
; 6(7)2021 04 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33651713
16.
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice.
Mol Ther
; 16(9): 1539-45, 2008 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-18665159
17.
Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate.
JACC Basic Transl Sci
; 4(7): 778-791, 2019 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-31998848
18.
Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances.
Biochim Biophys Acta
; 1772(2): 243-62, 2007 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-17064882
19.
Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
Nat Commun
; 8: 16007, 2017 06 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-28643790
20.
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
Nat Commun
; 8: 14454, 2017 02 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-28195574