Detalhe da pesquisa
1.
Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 119(9)2022 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35193974
2.
eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.
Nucleic Acids Res
; 49(W1): W193-W198, 2021 07 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34104972
3.
Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy.
Proc Natl Acad Sci U S A
; 117(28): 16509-16515, 2020 07 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-32601200
4.
DUX4 Transcript Knockdown with Antisense 2'-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy.
Mol Ther
; 29(2): 848-858, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33068777
5.
[Busulfan/thiotepa followed by autologous peripheral blood stem cell transplantation for refractory diffuse large B-cell lymphoma accompanied by hypopyon].
Rinsho Ketsueki
; 63(10): 1409-1414, 2022.
Artigo
em Japonês
| MEDLINE | ID: mdl-36351648
6.
Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform.
Int J Mol Sci
; 22(23)2021 Nov 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-34884423
7.
Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene.
Mol Ther
; 27(11): 2005-2017, 2019 11 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-31416775
8.
Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates.
Mol Ther
; 27(1): 76-86, 2019 01 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30448197
9.
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 114(16): 4213-4218, 2017 04 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-28373570
10.
Membrane Repair Deficit in Facioscapulohumeral Muscular Dystrophy.
Int J Mol Sci
; 21(15)2020 Aug 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32759720
11.
Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.
Mol Ther
; 25(11): 2561-2572, 2017 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28865998
12.
Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy.
J Hum Genet
; 62(4): 459-463, 2017 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-27974813
13.
Organ-specific gene expression: the bHLH protein Sage provides tissue specificity to Drosophila FoxA.
Development
; 140(10): 2160-71, 2013 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-23578928
14.
The PAF1 complex is involved in embryonic epidermal morphogenesis in Caenorhabditis elegans.
Dev Biol
; 391(1): 43-53, 2014 Jul 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-24721716
15.
Successful treatment with blinatumomab for acute lymphoblastic leukemia in an older adult patient complicated with hepatocarcinoma.
Leuk Res Rep
; 21: 100413, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38348414
16.
Drosophila as a model for epithelial tube formation.
Dev Dyn
; 241(1): 119-35, 2012 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-22083894
17.
Gilteritinib as Bridging and Posttransplant Maintenance for Relapsed Acute Myeloid Leukemia with FLT3-ITD Mutation Accompanied by Extramedullary Disease in Elderly.
Case Rep Hematol
; 2023: 7164742, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37662831
18.
DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.
JCI Insight
; 8(5)2023 03 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36719755
19.
Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation.
Nucleic Acid Ther
; 32(3): 185-193, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35085461
20.
Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting.
Methods Mol Biol
; 2224: 203-214, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-33606217