A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse von Porensky, Paul N., Mitrpant, Chalermchai, McGovern, Vicki L., Bevan, Adam K., Foust, Kevin D., Kaspar, Brain K., Wilton, Stephen D., Burghes, Arthur H.M.

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MicroRNAs Regulate Tumor Angiogenesis Modulated by Endothelial Progenitor Cells von PLUMMER, Prue N, FREEMAN, Ruth, FERRO, Vito, MCMILLAN, Nigel A. J, SWARBRICK, Alexander, MITTAL, Vivek, MELLICK, Albert S, TAFT, Ryan J, VIDER, Jelena, SAX, Michael, UMER, Brittany A, DINGCHENG GAO, JOHNS, Christopher, MATTICK, John S, WILTON, Stephen D

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Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro von Le, Bao T., Adams, Abbie M., Fletcher, Susan, Wilton, Stephen D., Veedu, Rakesh N.

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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse von Lu, Qi Long, Partridge, Terence A, Mann, Christopher J, Lou, Fang, Bou-Gharios, George, Morris, Glenn E, Xue, Shao-an, Fletcher, Sue, Wilton, Stephen D

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Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal von Pigozzo, Sarah R, Da Re, Lorena, Romualdi, Chiara, Mazzara, Pietro G, Galletta, Eva, Fletcher, Sue, Wilton, Stephen D, Vitiello, Libero

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Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency von Shaw, Nicole C, Kicic, Anthony, Fletcher, Sue, Wilton, Stephen D, Stick, Stephen M, Schultz, André

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A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice von Zhou, Haiyan, Janghra, Narinder, Mitrpant, Chalermchai, Dickinson, Rachel L, Anthony, Karen, Price, Loren, Eperon, Ian C, Wilton, Stephen D, Morgan, Jennifer, Muntoni, Francesco

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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides von Wilton, Stephen D, Lloyd, Frances, Carville, Kylie, Fletcher, Sue, Honeyman, Kaite, Agrawal, Sudhir, Kole, Ryszard

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Smart functional nucleic acid chimeras: Enabling tissue specific RNA targeting therapy von Aaldering, Lukas J, Tayeb, Hossam, Krishnan, Shilpa, Fletcher, Susan, Wilton, Stephen D, Veedu, Rakesh N

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Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle von Gebski, Bianca L., Mann, Chrisopher J., Fletcher, Susan, Wilton, Stephen D.

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Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the mdx Mouse von Mann, Christopher J., Honeyman, Kaite, Cheng, Andy J., Ly, Tina, Lloyd, Frances, Fletcher, Sue, Morgan, Jennifer E., Partridge, Terry A., Wilton, Stephen D.

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Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy von Mann, Christopher J., Honeyman, Kaite, McClorey, Graham, Fletcher, Sue, Wilton, Stephen D.

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Molecular analysis of a spontaneous dystrophin `knockout' dog von Schatzberg, Scott J, Olby, Natasha J, Breen, Matthew, Anderson, Louise V.B, Langford, Cordelia F, Dickens, Helen F, Wilton, Stephen D, Zeiss, Caroline J, Binns, Matthew M, Kornegay, Joe N, Morris, Glenn E, Sharp, Nicholas J.H

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Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse von Fletcher, Sue, Honeyman, Kaite, Fall, Abbie M, Harding, Penny L, Johnsen, Russell D, Steinhaus, Joshua P, Moulton, Hong M, Iversen, Patrick L, Wilton, Stephen D

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Antisense suppression of donor splice site mutations in the dystrophin gene transcript von Fletcher, Sue, Meloni, Penny L., Johnsen, Russell D., Wong, Brenda L., Muntoni, Francesco, Wilton, Stephen D.

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Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes von Bellgard, Matthew I., Sleeman, Mark W., Guerrero, Felix D., Fletcher, Sue, Baynam, Gareth, Goldblatt, Jack, Rubinstein, Yaffa, Bell, Callum, Groft, Stephen, Barrero, Roberto, Bittles, Alan H., Wilton, Stephen D., Mason, Christopher E., Weeramanthri, Tarun

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Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis von Laws, Nicola, Cornford-Nairn, Renee A, Irwin, Nicole, Johnsen, Russell, Fletcher, Susan, Wilton, Stephen D, Hoey, Andrew J

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A mutation in the α tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy von Laing, Nigel G, Akkari, Patrick A, Love, Donald R, Watkins, Hugh, Wilton, Stephen D, Dorosz, Shellie, Kneebone, Chris, White, Sue, Haan, Eric, Boundy, Karyn, Blumbergs, Peter

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Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene von Errington, Stephen J., Mann, Christopher J., Fletcher, Sue, Wilton, Stephen D.

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Terminal antisense oligonucleotide modifications can enhance induced exon skipping von Gebski, Bijanka L., Errington, Stephen J., Johnsen, Russell D., Fletcher, Susan, Wilton, Stephen D.

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