Detalhe da pesquisa
1.
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery.
Cell
; 186(10): 2062-2077.e17, 2023 05 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37075755
2.
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery.
Cell
; 186(16): 3520, 2023 Aug 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37541201
3.
In vivo selection of synthetic nucleocapsids for tissue targeting.
Proc Natl Acad Sci U S A
; 120(46): e2306129120, 2023 Nov 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37939083
4.
Duchenne muscular dystrophy models show their age.
Cell
; 143(7): 1040-2, 2010 Dec 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-21183068
5.
Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model.
Mol Ther
; 30(6): 2176-2185, 2022 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35143959
6.
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
7.
Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.
Mol Ther
; 29(3): 1070-1085, 2021 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33160075
8.
AAV-based gene therapies for the muscular dystrophies.
Hum Mol Genet
; 28(R1): R102-R107, 2019 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31238336
9.
Monitoring disease activity noninvasively in the mdx model of Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 115(30): 7741-7746, 2018 07 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-29987034
10.
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy.
Proc Natl Acad Sci U S A
; 115(39): E9182-E9191, 2018 09 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-30181272
11.
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
12.
Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins.
Hum Mol Genet
; 27(12): 2090-2100, 2018 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29618008
13.
A Boost for Muscle with Gene Therapy.
N Engl J Med
; 386(12): 1184-1186, 2022 Mar 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-35320650
14.
Development of Novel Micro-dystrophins with Enhanced Functionality.
Mol Ther
; 27(3): 623-635, 2019 03 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30718090
15.
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
16.
Progress toward Gene Therapy for Duchenne Muscular Dystrophy.
Mol Ther
; 25(5): 1125-1131, 2017 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28416280
17.
Muscle structure influences utrophin expression in mdx mice.
PLoS Genet
; 10(6): e1004431, 2014 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-24922526
18.
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
19.
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
20.
Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins.
Hum Mol Genet
; 27(15): 2773, 2018 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29901725