154 related articles for article (PubMed ID: 31873062)
21. Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Potter RA; Griffin DA; Sondergaard PC; Johnson RW; Pozsgai ER; Heller KN; Peterson EL; Lehtimäki KK; Windish HP; Mittal PJ; Albrecht DE; Mendell JR; Rodino-Klapac LR
Hum Gene Ther; 2018 Jul; 29(7):749-762. PubMed ID: 28707952
[TBL] [Abstract][Full Text] [Related]
22. An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.
Rodrigues M; Yokota T
Methods Mol Biol; 2018; 1828():31-55. PubMed ID: 30171533
[TBL] [Abstract][Full Text] [Related]
23. Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' Cells.
Barthélémy F; Blouin C; Wein N; Mouly V; Courrier S; Dionnet E; Kergourlay V; Mathieu Y; Garcia L; Butler-Browne G; Lamaze C; Lévy N; Krahn M; Bartoli M
J Neuromuscul Dis; 2015 Sep; 2(3):281-290. PubMed ID: 27858744
[TBL] [Abstract][Full Text] [Related]
24. 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]
25. Dysferlin Gene Mutation Spectrum in a Large Cohort of Chinese Patients with Dysferlinopathy.
Jin SQ; Yu M; Zhang W; Lyu H; Yuan Y; Wang ZX
Chin Med J (Engl); 2016 Oct; 129(19):2287-93. PubMed ID: 27647186
[TBL] [Abstract][Full Text] [Related]
26. An in-frame pseudoexon activation caused by a novel deep-intronic variant in the dysferlin gene.
Sun C; Xie Z; Cong L; Xu Y; Liu Z
Ann Clin Transl Neurol; 2023 Feb; 10(2):292-296. PubMed ID: 36542547
[TBL] [Abstract][Full Text] [Related]
27. Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.
Llanga T; Nagy N; Conatser L; Dial C; Sutton RB; Hirsch ML
Mol Ther; 2017 Sep; 25(9):2150-2162. PubMed ID: 28629822
[TBL] [Abstract][Full Text] [Related]
28. Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.
Wyatt EJ; Demonbreun AR; Kim EY; Puckelwartz MJ; Vo AH; Dellefave-Castillo LM; Gao QQ; Vainzof M; Pavanello RCM; Zatz M; McNally EM
JCI Insight; 2018 May; 3(9):. PubMed ID: 29720576
[TBL] [Abstract][Full Text] [Related]
29. AON-Mediated Exon Skipping to Bypass Protein Truncation in Retinal Dystrophies Due to the Recurrent
Barny I; Perrault I; Michel C; Goudin N; Defoort-Dhellemmes S; Ghazi I; Kaplan J; Rozet JM; Gerard X
Genes (Basel); 2019 May; 10(5):. PubMed ID: 31091803
[TBL] [Abstract][Full Text] [Related]
30. Identification and characterisation of human dysferlin transcript variants: implications for dysferlin mutational screening and isoforms.
Pramono ZA; Tan CL; Seah IA; See JS; Kam SY; Lai PS; Yee WC
Hum Genet; 2009 May; 125(4):413-20. PubMed ID: 19221801
[TBL] [Abstract][Full Text] [Related]
31. Private dysferlin exon skipping mutation (c.5492G>A) with a founder effect reveals further alternative splicing involving exons 49-51.
Santos R; Oliveira J; Vieira E; Coelho T; Carneiro AL; Evangelista T; Dias C; Fortuna A; Geraldo A; Negrão L; Guimarães A; Bronze-da-Rocha E
J Hum Genet; 2010 Aug; 55(8):546-9. PubMed ID: 20535123
[TBL] [Abstract][Full Text] [Related]
32. Genetic variability in Iranian limb-girdle muscular dystrophy type 2B patients: An evidence of a founder effect.
Mojbafan M; Tina S; Zafarghandi Motlagh F; Surguchov A; Nilipour Y; Zeinali S
Mol Genet Genomic Med; 2019 Dec; 7(12):e1029. PubMed ID: 31693312
[TBL] [Abstract][Full Text] [Related]
33. Exclusion of mutations in the dysferlin alternative exons 1 of DYSF-v1, 5a, and 40a in a cohort of 26 patients.
Krahn M; Labelle V; Borges A; Bartoli M; Lévy N
Genet Test Mol Biomarkers; 2010 Feb; 14(1):153-4. PubMed ID: 19929428
[TBL] [Abstract][Full Text] [Related]
34. The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments.
Anwar S; Yokota T
Biomolecules; 2024 Feb; 14(3):. PubMed ID: 38540676
[TBL] [Abstract][Full Text] [Related]
35. Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.
van Putten M; Tanganyika-de Winter C; Bosgra S; Aartsma-Rus A
Nucleic Acid Ther; 2019 Apr; 29(2):92-103. PubMed ID: 30672725
[TBL] [Abstract][Full Text] [Related]
36. 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]
37. 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]
38. The assembly and evaluation of antisense oligonucleotides applied in exon skipping for titin-based mutations in dilated cardiomyopathy.
Hahn JK; Neupane B; Pradhan K; Zhou Q; Testa L; Pelzl L; Maleck C; Gawaz M; Gramlich M
J Mol Cell Cardiol; 2019 Jun; 131():12-19. PubMed ID: 30998980
[TBL] [Abstract][Full Text] [Related]
39. Minimal expression of dysferlin prevents development of dysferlinopathy in dysferlin exon 40a knockout mice.
Yasa J; Reed CE; Bournazos AM; Evesson FJ; Pang I; Graham ME; Wark JR; Nijagal B; Kwan KH; Kwiatkowski T; Jung R; Weisleder N; Cooper ST; Lemckert FA
Acta Neuropathol Commun; 2023 Jan; 11(1):15. PubMed ID: 36653852
[TBL] [Abstract][Full Text] [Related]
40. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.
Aartsma-Rus A; Fokkema I; Verschuuren J; Ginjaar I; van Deutekom J; van Ommen GJ; den Dunnen JT
Hum Mutat; 2009 Mar; 30(3):293-9. PubMed ID: 19156838
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]