Detalhe da pesquisa
1.
PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice.
EMBO Mol Med
; 15(11): e17683, 2023 11 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37724723
2.
Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
Mol Ther
; 19(7): 1295-303, 2011 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-21505427
3.
Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy.
JCI Insight
; 7(24)2022 12 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-36346674
4.
Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO.
Mol Ther
; 18(10): 1822-9, 2010 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-20700113
5.
Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy.
JCI Insight
; 6(13)2021 07 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34236053
6.
Profiling of Extracellular Small RNAs Highlights a Strong Bias towards Non-Vesicular Secretion.
Cells
; 10(6)2021 06 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34207405
7.
Dystrophin involvement in peripheral circadian SRF signalling.
Life Sci Alliance
; 4(10)2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34389686
8.
From gut to brain: bioencapsulated therapeutic protein reduces amyloid load upon oral delivery.
Mol Ther
; 22(3): 485-486, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24584078
9.
Exosomes mediate sensory hair cell protection in the inner ear.
J Clin Invest
; 130(5): 2657-2672, 2020 05 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32027617
10.
Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.
J Clin Invest
; 129(11): 4739-4744, 2019 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31479430
11.
Intranasal exosomes for treatment of neuroinflammation? Prospects and limitations.
Mol Ther
; 19(10): 1754-6, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-21964306
12.
Lipid-based Transfection Reagents Exhibit Cryo-induced Increase in Transfection Efficiency.
Mol Ther Nucleic Acids
; 5: e290, 2016 Mar 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27111416
13.
Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency.
Mol Ther Nucleic Acids
; 2: e124, 2013 Sep 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-24064708
14.
Small RNA-Mediated Epigenetic Myostatin Silencing.
Mol Ther Nucleic Acids
; 1: e23, 2012 May 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23344003
15.
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.
Mol Ther Nucleic Acids
; 1: e38, 2012 Aug 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-23344180
16.
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.
Mol Ther Nucleic Acids
; 1: e62, 2012 Dec 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-23250360
17.
PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy.
Curr Opin Mol Ther
; 12(4): 478-86, 2010 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-20677099
18.
Oligonucleotide therapies: the future of amyotrophic lateral sclerosis treatment?
Neurodegener Dis Manag
; 5(2): 93-5, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-25894871
19.
Natural Antisense Makes Sense for Gene-specific Activation in Brain.
Mol Ther Nucleic Acids
; 1: e24, 2012 May 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23344004
20.
Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA.
Mol Ther Nucleic Acids
; 1: e27, 2012 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-23344079