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Results: 1 to 20 of 112

1.

Antisense oligonucleotide induction of progerin in human myogenic cells.

Luo YB, Mitrpant C, Adams AM, Johnsen RD, Fletcher S, Mastaglia FL, Wilton SD.

PLoS One. 2014 Jun 3;9(6):e98306. doi: 10.1371/journal.pone.0098306. eCollection 2014.

PMID:
24892300
[PubMed - in process]
Free PMC Article
2.

Dystrophin as a therapeutic biomarker: are we ignoring data from the past?

Wilton SD, Fletcher S, Flanigan KM.

Neuromuscul Disord. 2014 Jun;24(6):463-6. doi: 10.1016/j.nmd.2014.03.007. Epub 2014 Mar 22. No abstract available.

PMID:
24767581
[PubMed - in process]
3.

Targeted exon skipping to correct exon duplications in the dystrophin gene.

Greer KL, Lochmüller H, Flanigan K, Fletcher S, Wilton SD.

Mol Ther Nucleic Acids. 2014 Mar 18;3:e155. doi: 10.1038/mtna.2014.8.

PMID:
24643206
[PubMed]
Free PMC Article
4.

Antisense suppression of donor splice site mutations in the dystrophin gene transcript.

Fletcher S, Meloni PL, Johnsen RD, Wong BL, Muntoni F, Wilton SD.

Mol Genet Genomic Med. 2013 Sep;1(3):162-73. doi: 10.1002/mgg3.19. Epub 2013 Jun 13.

PMID:
24498612
[PubMed]
Free PMC Article
5.

Normal and aberrant splicing of LMNA.

Luo YB, Mastaglia FL, Wilton SD.

J Med Genet. 2014 Apr;51(4):215-23. doi: 10.1136/jmedgenet-2013-102119. Epub 2014 Jan 23.

PMID:
24459210
[PubMed - in process]
6.

Investigation of age-related changes in LMNA splicing and expression of progerin in human skeletal muscles.

Luo YB, Mitrpant C, Johnsen RD, Fabian VA, Fletcher S, Mastaglia FL, Wilton SD.

Int J Clin Exp Pathol. 2013 Nov 15;6(12):2778-86. eCollection 2013.

PMID:
24294364
[PubMed - in process]
Free PMC Article
7.

Primary over-expression of AβPP in muscle does not lead to the development of inclusion body myositis in a new lineage of the MCK-AβPP transgenic mouse.

Luo YB, Johnsen RD, Griffiths L, Needham M, Fabian VA, Fletcher S, Wilton SD, Mastaglia FL.

Int J Exp Pathol. 2013 Dec;94(6):418-25. doi: 10.1111/iep.12048. Epub 2013 Oct 31.

PMID:
24205796
[PubMed - indexed for MEDLINE]
8.

Complement-mediated muscle cell lysis: a possible mechanism of myonecrosis in anti-SRP associated necrotizing myopathy (ASANM).

Rojana-udomsart A, Mitrpant C, Bundell C, Price L, Luo YB, Fabian V, Wilton SD, Hollingsworth P, Mastaglia FL.

J Neuroimmunol. 2013 Nov 15;264(1-2):65-70. doi: 10.1016/j.jneuroim.2013.08.008. Epub 2013 Aug 30.

PMID:
24041831
[PubMed - indexed for MEDLINE]
9.

Investigation of splicing changes and post-translational processing of LMNA in sporadic inclusion body myositis.

Luo YB, Mitrpant C, Johnsen R, Fabian V, Needham M, Fletcher S, Wilton SD, Mastaglia FL.

Int J Clin Exp Pathol. 2013 Aug 15;6(9):1723-33. eCollection 2013.

PMID:
24040437
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal.

Pigozzo SR, Da Re L, Romualdi C, Mazzara PG, Galletta E, Fletcher S, Wilton SD, Vitiello L.

PLoS One. 2013 Aug 28;8(8):e72147. doi: 10.1371/journal.pone.0072147. eCollection 2013.

PMID:
24015212
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AH.

PLoS One. 2013 Apr 22;8(4):e62114. doi: 10.1371/journal.pone.0062114. Print 2013.

PMID:
23630626
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.

PMID:
23369965
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Targeted exon skipping to address "leaky" mutations in the dystrophin gene.

Fletcher S, Adkin CF, Meloni P, Wong B, Muntoni F, Kole R, Fragall C, Greer K, Johnsen R, Wilton SD.

Mol Ther Nucleic Acids. 2012 Oct 16;1:e48. doi: 10.1038/mtna.2012.40.

PMID:
23344648
[PubMed]
Free PMC Article
14.

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice.

Zhou H, Janghra N, Mitrpant C, Dickinson RL, Anthony K, Price L, Eperon IC, Wilton SD, Morgan J, Muntoni F.

Hum Gene Ther. 2013 Mar;24(3):331-42. doi: 10.1089/hum.2012.211. Epub 2013 Mar 6.

PMID:
23339722
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

A cell-based high-throughput screening assay for posttranscriptional utrophin upregulation.

Moorwood C, Soni N, Patel G, Wilton SD, Khurana TS.

J Biomol Screen. 2013 Apr;18(4):400-6. doi: 10.1177/1087057112465648. Epub 2012 Oct 30.

PMID:
23112083
[PubMed - indexed for MEDLINE]
16.

Analysis of HLA-DRB3 alleles and supertypical genotypes in the MHC Class II region in sporadic inclusion body myositis.

Rojana-udomsart A, Mitrpant C, James I, Witt C, Needham M, Day T, Kiers L, Corbett A, Martinez P, Wilton SD, Mastaglia FL.

J Neuroimmunol. 2013 Jan 15;254(1-2):174-7. doi: 10.1016/j.jneuroim.2012.09.003. Epub 2012 Sep 23.

PMID:
23010279
[PubMed - indexed for MEDLINE]
17.

MicroRNAs regulate tumor angiogenesis modulated by endothelial progenitor cells.

Plummer PN, Freeman R, Taft RJ, Vider J, Sax M, Umer BA, Gao D, Johns C, Mattick JS, Wilton SD, Ferro V, McMillan NA, Swarbrick A, Mittal V, Mellick AS.

Cancer Res. 2013 Jan 1;73(1):341-52. doi: 10.1158/0008-5472.CAN-12-0271. Epub 2012 Jul 25.

PMID:
22836757
[PubMed - indexed for MEDLINE]
Free Article
18.

Co-regulation of survival of motor neuron and Bcl-xL expression: implications for neuroprotection in spinal muscular atrophy.

Anderton RS, Price LL, Turner BJ, Meloni BP, Mitrpant C, Mastaglia FL, Goh C, Wilton SD, Boulos S.

Neuroscience. 2012 Sep 18;220:228-36. doi: 10.1016/j.neuroscience.2012.06.042. Epub 2012 Jun 23.

PMID:
22732506
[PubMed - indexed for MEDLINE]
19.

Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements.

Barrett LW, Fletcher S, Wilton SD.

Cell Mol Life Sci. 2012 Nov;69(21):3613-34. doi: 10.1007/s00018-012-0990-9. Epub 2012 Apr 27. Review.

PMID:
22538991
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Optimizing splice-switching oligomer sequences using 2'-O-methyl phosphorothioate chemistry.

Adkin C, Fletcher S, Wilton SD.

Methods Mol Biol. 2012;867:169-88. doi: 10.1007/978-1-61779-767-5_11.

PMID:
22454061
[PubMed - indexed for MEDLINE]

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