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Results: 1 to 20 of 118

1.

A Registry Framework Enabling Patient-Centred Care.

Bellgard MI, Napier K, Render L, Radochonski M, Lamont L, Graham C, Wilton SD, Fletcher S, Goldblatt J, Hunter AA, Weeramanthri T.

Stud Health Technol Inform. 2015;214:8-14.

PMID:
26210411
2.

The emperor's new dystrophin: finding sense in the noise.

Wilton SD, Veedu RN, Fletcher S.

Trends Mol Med. 2015 Jul;21(7):417-26. doi: 10.1016/j.molmed.2015.04.006. Epub 2015 Jun 4. Review.

PMID:
26051381
3.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 May;21(5):537. doi: 10.1038/nm0515-537c. No abstract available.

PMID:
25951531
4.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 Apr;21(4):414. doi: 10.1038/nm0415-414b. No abstract available.

PMID:
25849273
5.

Smart functional nucleic acid chimeras: enabling tissue specific RNA targeting therapy.

Aaldering LJ, Tayeb H, Krishnan S, Fletcher S, Wilton SD, Veedu RN.

RNA Biol. 2015;12(4):412-25. doi: 10.1080/15476286.2015.1017234.

PMID:
25849197
6.

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2014 Sep;20(9):992-1000. doi: 10.1038/nm.3628. Epub 2014 Aug 10. Erratum in: Nat Med. 2015 May;21(5):537. Nat Med. 2015 May;21(5):537. Brioschi, Simona [added]; Bovolenta, Matteo [added]; Neri, Marcella [added]. Nat Med. 2015 Apr;21(4):414.

7.

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.

8.

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
9.

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.

10.

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.

11.

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. Review.

PMID:
24459210
12.

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.

13.

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.

14.

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
15.

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.

16.

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.

17.

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.

18.

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.

19.

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.

20.

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.

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