Detalhe da pesquisa
1.
Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.
Int J Mol Sci
; 24(3)2023 Jan 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-36769018
2.
Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.
Mol Ther
; 28(12): 2527-2539, 2020 12 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33171139
3.
Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7.
Nucleic Acids Res
; 45(13): 7870-7885, 2017 Jul 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-28575281
4.
C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.
Brain
; 140(4): 887-897, 2017 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28334866
5.
Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles.
Mol Ther
; 25(7): 1580-1587, 2017 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28392161
6.
Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.
Methods Mol Biol
; 2587: 209-237, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36401033
7.
A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles.
Nanoscale Adv
; 5(11): 2941-2949, 2023 May 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-37260495
8.
Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.
Mol Ther Nucleic Acids
; 34: 102024, 2023 Dec 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37744174
9.
Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.
Mol Ther Nucleic Acids
; 27: 1146-1155, 2022 Mar 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35282418
10.
Heterogeneous distribution of SNPs in the human genome: microsatellites as predictors of nucleotide diversity and divergence.
Genomics
; 95(3): 151-9, 2010 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-20026267
11.
Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.
Mol Ther Nucleic Acids
; 26: 174-191, 2021 Dec 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34513303
12.
Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion.
Methods Mol Biol
; 2176: 185-208, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32865792
13.
A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN.
Cells
; 9(11)2020 11 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33153033
14.
Evidence for nonindependent evolution of adjacent microsatellites in the human genome.
J Mol Evol
; 68(2): 160-70, 2009 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-19184164
15.
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
16.
Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.
Eur J Hum Genet
; 24(2): 271-6, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25990798
17.
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.
Sci Rep
; 5: 8986, 2015 Mar 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-25758104
18.
Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.
Mol Ther Nucleic Acids
; 3: e212, 2014 Nov 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-25423116
19.
Cross-talking noncoding RNAs contribute to cell-specific neurodegeneration in SCA7.
Nat Struct Mol Biol
; 21(11): 955-961, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25306109
20.
Epigenetics and ncRNAs in brain function and disease: mechanisms and prospects for therapy.
Neurotherapeutics
; 10(4): 621-31, 2013 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-24068583