287 related articles for article (PubMed ID: 26427454)
21. Splicing intervention for Duchenne muscular dystrophy.
McClorey G; Fletcher S; Wilton S
Curr Opin Pharmacol; 2005 Oct; 5(5):529-34. PubMed ID: 16085461
[TBL] [Abstract][Full Text] [Related]
22. Development and Use of Cellular Systems to Assess and Correct Splicing Defects.
Suárez-Herrera N; Tomkiewicz TZ; Garanto A; Collin RWJ
Methods Mol Biol; 2022; 2434():145-165. PubMed ID: 35213015
[TBL] [Abstract][Full Text] [Related]
23. Molecular genetic testing in clinical diagnostic assessments that demonstrate correlations in patients with autosomal recessive inherited retinal dystrophy.
Liu X; Xiao J; Huang H; Guan L; Zhao K; Xu Q; Zhang X; Pan X; Gu S; Chen Y; Zhang J; Shen Y; Jiang H; Gao X; Kang X; Sheng X; Chen X; Zhao C
JAMA Ophthalmol; 2015 Apr; 133(4):427-36. PubMed ID: 25611614
[TBL] [Abstract][Full Text] [Related]
24. Splice-switching antisense oligonucleotides as therapeutic drugs.
Havens MA; Hastings ML
Nucleic Acids Res; 2016 Aug; 44(14):6549-63. PubMed ID: 27288447
[TBL] [Abstract][Full Text] [Related]
25. Gene Therapy for Inherited Retinal Disorders: Update on Clinical Trials.
Michalakis S; Gerhardt M; Rudolph G; Priglinger S; Priglinger C
Klin Monbl Augenheilkd; 2021 Mar; 238(3):272-281. PubMed ID: 33784790
[TBL] [Abstract][Full Text] [Related]
26. Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases.
Du L; Gatti RA
J Immunol Methods; 2011 Feb; 365(1-2):1-7. PubMed ID: 21147113
[TBL] [Abstract][Full Text] [Related]
27. Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases.
Veltrop M; Aartsma-Rus A
Exp Cell Res; 2014 Jul; 325(1):50-5. PubMed ID: 24486759
[TBL] [Abstract][Full Text] [Related]
28. Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in.
Popplewell L; Koo T; Leclerc X; Duclert A; Mamchaoui K; Gouble A; Mouly V; Voit T; Pâques F; Cédrone F; Isman O; Yáñez-Muñoz RJ; Dickson G
Hum Gene Ther; 2013 Jul; 24(7):692-701. PubMed ID: 23790397
[TBL] [Abstract][Full Text] [Related]
29. The new landscape of retinal gene therapy.
Ku CA; Pennesi ME
Am J Med Genet C Semin Med Genet; 2020 Sep; 184(3):846-859. PubMed ID: 32888388
[TBL] [Abstract][Full Text] [Related]
30. In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.
Blázquez L; Aiastui A; Goicoechea M; Martins de Araujo M; Avril A; Beley C; García L; Valcárcel J; Fortes P; López de Munain A
Hum Mutat; 2013 Oct; 34(10):1387-95. PubMed ID: 23864287
[TBL] [Abstract][Full Text] [Related]
31. Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.
Jirka SM; Heemskerk H; Tanganyika-de Winter CL; Muilwijk D; Pang KH; de Visser PC; Janson A; Karnaoukh TG; Vermue R; 't Hoen PA; van Deutekom JC; Aguilera B; Aartsma-Rus A
Nucleic Acid Ther; 2014 Feb; 24(1):25-36. PubMed ID: 24320790
[TBL] [Abstract][Full Text] [Related]
32. Induced dystrophin exon skipping in human muscle explants.
McClorey G; Fall AM; Moulton HM; Iversen PL; Rasko JE; Ryan M; Fletcher S; Wilton SD
Neuromuscul Disord; 2006 Oct; 16(9-10):583-90. PubMed ID: 16919955
[TBL] [Abstract][Full Text] [Related]
33. Gene supplementation therapy for recessive forms of inherited retinal dystrophies.
Smith AJ; Bainbridge JW; Ali RR
Gene Ther; 2012 Feb; 19(2):154-61. PubMed ID: 22033465
[TBL] [Abstract][Full Text] [Related]
34. Overview on AON design.
Aartsma-Rus A
Methods Mol Biol; 2012; 867():117-29. PubMed ID: 22454058
[TBL] [Abstract][Full Text] [Related]
35. Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid.
Huang D; Fletcher S; Wilton SD; Palmer N; McLenachan S; Mackey DA; Chen FK
Vision (Basel); 2017 Sep; 1(3):. PubMed ID: 31740647
[TBL] [Abstract][Full Text] [Related]
36. Making antisense of splicing.
Garcia-Blanco MA
Curr Opin Mol Ther; 2005 Oct; 7(5):476-82. PubMed ID: 16248283
[TBL] [Abstract][Full Text] [Related]
37. Modification of pre-mRNA processing: application to dystrophin expression.
Wilton SD; Fletcher S
Curr Opin Mol Ther; 2006 Apr; 8(2):130-5. PubMed ID: 16610765
[TBL] [Abstract][Full Text] [Related]
38. Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays.
Graham IR; Hill VJ; Manoharan M; Inamati GB; Dickson G
J Gene Med; 2004 Oct; 6(10):1149-58. PubMed ID: 15386737
[TBL] [Abstract][Full Text] [Related]
39. Improving the management of Inherited Retinal Dystrophies by targeted sequencing of a population-specific gene panel.
Bravo-Gil N; Méndez-Vidal C; Romero-Pérez L; González-del Pozo M; Rodríguez-de la Rúa E; Dopazo J; Borrego S; Antiñolo G
Sci Rep; 2016 Apr; 6():23910. PubMed ID: 27032803
[TBL] [Abstract][Full Text] [Related]
40. [Gene Replacement Therapy for Inherited Retinal Dystrophies].
Mühlfriedel R; Sothilingam V; Tanimoto N; Seeliger MW
Klin Monbl Augenheilkd; 2017 Mar; 234(3):320-328. PubMed ID: 28355661
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
