274 related articles for article (PubMed ID: 12077324)
1. Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells.
De Angelis FG; Sthandier O; Berarducci B; Toso S; Galluzzi G; Ricci E; Cossu G; Bozzoni I
Proc Natl Acad Sci U S A; 2002 Jul; 99(14):9456-61. PubMed ID: 12077324
[TBL] [Abstract][Full Text] [Related]
2. Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.
Incitti T; De Angelis FG; Cazzella V; Sthandier O; Pinnarò C; Legnini I; Bozzoni I
Mol Ther; 2010 Sep; 18(9):1675-82. PubMed ID: 20551908
[TBL] [Abstract][Full Text] [Related]
3. Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression.
Lee JJA; Saito T; Duddy W; Takeda S; Yokota T
Methods Mol Biol; 2018; 1828():141-150. PubMed ID: 30171539
[TBL] [Abstract][Full Text] [Related]
4. Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
Goyenvalle A; Davies KE
Methods Mol Biol; 2011; 709():179-96. PubMed ID: 21194028
[TBL] [Abstract][Full Text] [Related]
5. Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.
Anthony K; Arechavala-Gomeza V; Ricotti V; Torelli S; Feng L; Janghra N; Tasca G; Guglieri M; Barresi R; Armaroli A; Ferlini A; Bushby K; Straub V; Ricci E; Sewry C; Morgan J; Muntoni F
JAMA Neurol; 2014 Jan; 71(1):32-40. PubMed ID: 24217213
[TBL] [Abstract][Full Text] [Related]
6. In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient.
Nakamura A; Aoki Y; Tsoumpra M; Yokota T; Takeda S
Methods Mol Biol; 2018; 1828():151-163. PubMed ID: 30171540
[TBL] [Abstract][Full Text] [Related]
7. Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.
Hanson B; Wood MJA; Roberts TC
RNA Biol; 2021 Jul; 18(7):1048-1062. PubMed ID: 33472516
[TBL] [Abstract][Full Text] [Related]
8. Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.
Echigoya Y; Lim KRQ; Trieu N; Bao B; Miskew Nichols B; Vila MC; Novak JS; Hara Y; Lee J; Touznik A; Mamchaoui K; Aoki Y; Takeda S; Nagaraju K; Mouly V; Maruyama R; Duddy W; Yokota T
Mol Ther; 2017 Nov; 25(11):2561-2572. PubMed ID: 28865998
[TBL] [Abstract][Full Text] [Related]
9. In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
Wang Q; Yin H; Camelliti P; Betts C; Moulton H; Lee H; Saleh AF; Gait MJ; Wood MJ
J Gene Med; 2010 Apr; 12(4):354-64. PubMed ID: 20235089
[TBL] [Abstract][Full Text] [Related]
10. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells.
van Deutekom JC; Bremmer-Bout M; Janson AA; Ginjaar IB; Baas F; den Dunnen JT; van Ommen GJ
Hum Mol Genet; 2001 Jul; 10(15):1547-54. PubMed ID: 11468272
[TBL] [Abstract][Full Text] [Related]
11. Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping.
Bertoni C; Lau C; Rando TA
Hum Mol Genet; 2003 May; 12(10):1087-99. PubMed ID: 12719373
[TBL] [Abstract][Full Text] [Related]
12. Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.
Echigoya Y; Yokota T
Nucleic Acid Ther; 2014 Feb; 24(1):57-68. PubMed ID: 24380394
[TBL] [Abstract][Full Text] [Related]
13. Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.
Anthony K; Cirak S; Torelli S; Tasca G; Feng L; Arechavala-Gomeza V; Armaroli A; Guglieri M; Straathof CS; Verschuuren JJ; Aartsma-Rus A; Helderman-van den Enden P; Bushby K; Straub V; Sewry C; Ferlini A; Ricci E; Morgan JE; Muntoni F
Brain; 2011 Dec; 134(Pt 12):3547-59. PubMed ID: 22102647
[TBL] [Abstract][Full Text] [Related]
14. Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy.
Béroud C; Tuffery-Giraud S; Matsuo M; Hamroun D; Humbertclaude V; Monnier N; Moizard MP; Voelckel MA; Calemard LM; Boisseau P; Blayau M; Philippe C; Cossée M; Pagès M; Rivier F; Danos O; Garcia L; Claustres M
Hum Mutat; 2007 Feb; 28(2):196-202. PubMed ID: 17041910
[TBL] [Abstract][Full Text] [Related]
15. U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping.
Brun C; Suter D; Pauli C; Dunant P; Lochmüller H; Burgunder JM; Schümperli D; Weis J
Cell Mol Life Sci; 2003 Mar; 60(3):557-66. PubMed ID: 12737315
[TBL] [Abstract][Full Text] [Related]
16. Designing Effective Antisense Oligonucleotides for Exon Skipping.
Shimo T; Maruyama R; Yokota T
Methods Mol Biol; 2018; 1687():143-155. PubMed ID: 29067661
[TBL] [Abstract][Full Text] [Related]
17. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.
Aartsma-Rus A; Bremmer-Bout M; Janson AA; den Dunnen JT; van Ommen GJ; van Deutekom JC
Neuromuscul Disord; 2002 Oct; 12 Suppl 1():S71-7. PubMed ID: 12206800
[TBL] [Abstract][Full Text] [Related]
18. Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice.
Hiller M; Spitali P; Datson N; Aartsma-Rus A
Methods Mol Biol; 2018; 1828():249-262. PubMed ID: 30171546
[TBL] [Abstract][Full Text] [Related]
19. A novel splicing silencer generated by DMD exon 45 deletion junction could explain upstream exon 44 skipping that modifies dystrophinopathy.
Dwianingsih EK; Malueka RG; Nishida A; Itoh K; Lee T; Yagi M; Iijima K; Takeshima Y; Matsuo M
J Hum Genet; 2014 Aug; 59(8):423-9. PubMed ID: 24871807
[TBL] [Abstract][Full Text] [Related]
20. Exons 45-55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells.
He M; Yokota T
Methods Mol Biol; 2023; 2640():313-325. PubMed ID: 36995604
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
