Detalhe da pesquisa
1.
Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies.
Neuropathol Appl Neurobiol
; 47(6): 711-723, 2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33999469
2.
Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy.
Mol Ther
; 20(2): 462-7, 2012 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-22086232
3.
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics.
Nucleic Acid Ther
; 33(4): 238-247, 2023 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37145922
4.
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides.
Nucleic Acid Ther
; 33(1): 1-16, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36579950
5.
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Mol Ther Nucleic Acids
; 34: 102066, 2023 Dec 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38034032
6.
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Lancet
; 378(9791): 595-605, 2011 Aug 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-21784508
7.
Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.
Brain
; 134(Pt 12): 3547-59, 2011 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-22102647
8.
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.
Mol Ther
; 19(2): 345-54, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-21102560
9.
Antisense RNA Therapeutics: A Brief Overview.
Methods Mol Biol
; 2434: 33-49, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35213008
10.
Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy.
Methods Mol Biol
; 2434: 217-233, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35213020
11.
miR-106b is a novel target to promote muscle regeneration and restore satellite stem cell function in injured Duchenne dystrophic muscle.
Mol Ther Nucleic Acids
; 29: 769-786, 2022 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36159592
12.
Researcher's Perceptions on Publishing "Negative" Results and Open Access.
Nucleic Acid Ther
; 31(3): 185-189, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32730128
13.
Special Issue "Genetic Advances in Neuromuscular Disorders: From Gene Identification to Gene Therapy".
Genes (Basel)
; 12(2)2021 02 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33567614
14.
Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening.
Sci Rep
; 11(1): 18188, 2021 09 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-34521928
15.
Delivery of oligonucleotide-based therapeutics: challenges and opportunities.
EMBO Mol Med
; 13(4): e13243, 2021 04 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33821570
16.
Joining European Scientific Forces to Face Pandemics.
Trends Microbiol
; 29(2): 92-97, 2021 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33288385
17.
The Biomarker Potential of miRNAs in Myotonic Dystrophy Type I.
J Clin Med
; 9(12)2020 Dec 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33291833
18.
An Overview of Alternative Splicing Defects Implicated in Myotonic Dystrophy Type I.
Genes (Basel)
; 11(9)2020 09 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-32971903
19.
Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology.
J Neuromuscul Dis
; 6(1): 147-159, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30614809
20.
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
PLoS One
; 13(10): e0204485, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-30278058