Detalhe da pesquisa
1.
Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2.mdx Mice.
Int J Mol Sci
; 25(11)2024 Jun 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38892298
2.
Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model.
Hum Mol Genet
; 30(15): 1398-1412, 2021 07 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33987655
3.
Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy.
Int J Mol Sci
; 24(9)2023 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37175881
4.
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model.
Gene Ther
; 29(9): 520-535, 2022 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-35105949
5.
G-quadruplex ligands mediate downregulation of DUX4 expression.
Nucleic Acids Res
; 48(8): 4179-4194, 2020 05 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32182342
6.
Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice.
Hum Mol Genet
; 28(18): 3091-3100, 2019 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-31179493
7.
Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9.
Mol Ther
; 26(6): 1529-1538, 2018 06 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29730196
8.
Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach.
Hum Mol Genet
; 25(8): 1468-78, 2016 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26787513
9.
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.
Nucleic Acids Res
; 44(22): 10929-10945, 2016 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27507886
10.
Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.
Mol Ther
; 23(8): 1341-1348, 2015 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-25959011
11.
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases.
Nucleic Acids Res
; 40(13): 6367-79, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22467209
12.
Expression of the Pro-Fibrotic Marker Periostin in a Mouse Model of Duchenne Muscular Dystrophy.
Biomedicines
; 12(1)2024 Jan 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-38255321
13.
Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD.
Methods Mol Biol
; 2587: 557-568, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36401050
14.
Novel Metabolomic Approach for Identifying Pathology-Specific Biomarkers in Rare Diseases: A Case Study in Oculopharyngeal Muscular Dystrophy (OPMD).
Metabolites
; 13(6)2023 Jun 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-37367926
15.
Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.
Mol Ther
; 19(1): 159-64, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-20924365
16.
Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy.
Molecules
; 17(2): 1138-48, 2012 Jan 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-22274137
17.
Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression.
Biomedicines
; 10(7)2022 Jul 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35884928
18.
Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment.
Methods Mol Biol
; 2434: 301-313, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35213026
19.
Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy.
Mol Ther Methods Clin Dev
; 25: 491-507, 2022 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35615709
20.
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy.
Hum Gene Ther
; 33(17-18): 923-935, 2022 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-35078334