Professor George Dickson

  1. 2019
  2. E-pub ahead of print

    Vaccination of macaques with DNA followed by adenoviral vectors encoding SIV gag alone delays infection by repeated mucosal challenge with SIV

    Almond, N., Berry, N., Stebbings, R., Preston, M., Ham, C., Page, M., Ferguson, D., Rose, N., Li, B., Mee, E. T., Hassall, M., Stahl-Hennig, C., Athanasopoulos, T., Papagatsias, T., Herath, S., Benlahrech, A., Dickson, G., Meiser, A. & Patterson, S., 14 Aug 2019, In : Journal of virology. p. 1-38 38 p.

    Research output: Contribution to journalArticle

  3. E-pub ahead of print

    Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1

    Malerba, A., Klein, P., Lu-Nguyen, N., Cappellari, O., Strings-Ufombah, V., Harbaran, S., Roelvink, P., Suhy, D., Trollet, C. & Dickson, G., 11 Jul 2019, In : Human Molecular Genetics. p. 1-24 24 p., ddz167.

    Research output: Contribution to journalArticle

  4. E-pub ahead of print

    Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice

    Lu-Nguyen, N., Ferry, A., Schnell, F. J., Hanson, G. J., Popplewell, L., Dickson, G. & Malerba, A., 10 Jun 2019, In : Human Molecular Genetics. ddz125.

    Research output: Contribution to journalArticle

  5. Published

    Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy

    Malerba, A., Roth, F., Harish, P., Dhiab, J., Lu-Nguyen, N., Cappellari, O., Jarmin, S., Mahoudeau, A., Ythier, V., Laine, J., Negroni, E., Abgueguen, E., Simonelig, M., Guedat, P., Mouly, V., Butler-Browne, G., Voisset, C., Dickson, G. & Trollet, C., 15 May 2019, In : Human Molecular Genetics. 28, 10, p. 1694-1708 15 p., ddz007.

    Research output: Contribution to journalArticle

  6. E-pub ahead of print

    Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)

    Harish, P., Malerba, A., Lu-Nguyen, N., Forrest, L., Cappellari, O., Roth, F., Trollet, C., Popplewell, L. & Dickson, J., 7 May 2019, In : Journal of Cachexia, Sarcopenia and Muscle. p. 1-11 11 p.

    Research output: Contribution to journalArticle

  7. Published

    Next-generation muscle-directed gene therapy by in silico vector design

    Sarcar, S., Tulalamba, W., Rincon, M. Y., Tipanee, J., Pham, H. Q., Evens, H., Boon, D., Samara-Kuko, E., Keyaerts, M., Loperfido, M., Berardi, E., Jarmin, S., In’t Veld, P., Dickson, G., Lahoutte, T., Sampaolesi, M., De Bleser, P., VandenDriessche, T. & Chuah, M. K., 30 Jan 2019, In : Nature Communications. 10, p. 1-16 16 p., 492.

    Research output: Contribution to journalArticle

  8. 2018
  9. Published

    A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures

    Hiller, M., Falzarano, M. S., Garcia-Jimenez, I., Sardone, V., Verheul, R. C., Popplewell, L., Anthony, K., Ruiz-Del-Yerro, E., Osman, H., Goeman, J. J., Mamchaoui, K., Dickson, G., Ferlini, A., Muntoni, F., Aartsma-Rus, A., Arechavala-Gomeza, V., Datson, N. A. & Spitali, P., 2 Oct 2018, In : PLoS ONE. 13, 10, p. 1-15 15 p., e0204485.

    Research output: Contribution to journalArticle

  10. E-pub ahead of print

    Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation

    Lu-Nguyen, N., Dickson, G. & Malerba, A., 1 Sep 2018, In : Methods in Molecular Biology. 1828, p. 343-354 12 p.

    Research output: Contribution to journalArticle

  11. Published

    Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

    Koo, T., Lu-Nguyen, N. B., Malerba, A., Kim, E., Kim, D., Cappellari, O., Cho, H-Y., Dickson, G., Popplewell, L. & Kim, J-S., 6 Jun 2018, In : Molecular Therapy. 26, 6, p. 1529-1538 10 p.

    Research output: Contribution to journalArticle

  12. Published

    Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy

    Benedetti, S., Uno, N., Hoshiya, H., Ragazzi, M., Ferrari, G., Kazuki, Y., Moyle, L. A., Tonlorenzi, R., Lombardo, A., Chaouch, S., Mouly, V., Moore, M., Popplewell, L., Kazuki, K., Katoh, M., Naldini, L., Dickson, G., Messina, G., Oshimura, M., Cossu, G. & 1 others, Tedesco, F. S., 1 Feb 2018, In : EMBO Reports. 10, 2, p. 254-275 22 p.

    Research output: Contribution to journalArticle

  13. 2017
  14. Published

    Synapsin I phosphorylation is dysregulated by beta-amyloid oligomers and restored by valproic acid

    Marsh, J., Bagol, S. H., Williams, R., Dickson, J. & Alifragis, P., Oct 2017, In : Neurobiology of Disease. 106, p. 63-75 13 p.

    Research output: Contribution to journalArticle

  15. Published

    Dysfunctional transcripts are formed by alternative polyadenylation in OPMD

    Raz, V., Dickson, J. & 't Hoen, P. A. C., 5 Sep 2017, In : Oncotarget. 8, 43, p. 73516-73528 13 p.

    Research output: Contribution to journalArticle

  16. Published

    Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

    Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., Allais, M., François, V., Dutilleul, M., Malerba, A., Koo, T., Thibaut, J-L., Matot, B., Devaux, M., Le Duff, J., Deschamps, J-Y., Barthelemy, I., Blot, S., Testault, I., Wahbi, K. & 15 others, Ederhy, S., Martin, S., Veron, P., Georger, C., Athanasopoulos, T., Masurier, C., Mingozzi, F., Carlier, P., Gjata, B., Hogrel, J-Y., Adjali, O., Mavilio, F., Voit, T., Moullier, P. & Dickson, G., 25 Jul 2017, In : Nature Communications. 8, p. 1-15 15 p., 16105.

    Research output: Contribution to journalArticle

  17. Published
  18. Published

    BNA 2017 Festival of Neuroscience: Abstract Book: Using in vitro systems to study the role of isoform-specific Apolipoprotein E processing in Alzheimer’s Disease

    Marsh, J., Dickson, J. & Alifragis, P., 7 Apr 2017, Brain and Neuroscience Advances. Dalley, J. (ed.). SAGE Publications Ltd, Vol. 1. p. 173 1 p.

    Research output: Chapter in Book/Report/Conference proceedingConference contribution

  19. Published

    PABPN1 gene therapy for oculopharyngeal muscular dystrophy

    Malerba, A., Klein, P., Bachtarzi, H., Jarmin, S. A., Cordova, G., Ferry, A., Strings, V., Espinoza, M. P., Mamchaoui, K., Blumen, S. C., St Guily, J. L., Mouly, V., Graham, M., Butler-Browne, G., Suhy, D. A., Trollet, C. & Dickson, G., 31 Mar 2017, In : Nature Communications. 8, p. 1-14 14 p., 14848.

    Research output: Contribution to journalArticle

  20. Published

    Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

    Lu-Nguyen, N., Malerba, A., Popplewell, L., Schnell, F., Hanson, G. & Dickson, G., 17 Mar 2017, In : Molecular therapy. Nucleic acids. 6, p. 15-28 14 p.

    Research output: Contribution to journalArticle

  21. Published

    Chimeric Trojan Protein Insertion in Lentiviral Membranes Makes Lentiviruses Susceptible to Neutralization by Anti-Tetanus Serum Antibodies

    Le-Heron, A., Patterson, S., Yáñez-Muñoz, R. J. & Dickson, G., 2017, In : Human Gene Therapy. 28, 3, p. 242-254 13 p.

    Research output: Contribution to journalArticle

  22. 2016
  23. Published

    Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing

    Klein, P., Oloko, M., Roth, F., Montel, V., Malerba, A., Jarmin, S., Gidaro, T., Popplewell, L., Perie, S., Lacau St Guily, J., de la Grange, P., Antoniou, M. N., Dickson, G., Butler-Browne, G., Bastide, B., Mouly, V. & Trollet, C., Dec 2016, In : Nucleic Acids Research. 44, 22, p. 10929–10945 17 p.

    Research output: Contribution to journalArticle

  24. Published

    Cytokine genes as potential biomarkers for muscle weakness in OPMD

    Riaz, M., Raz, Y., van der Slujis, B., Dickson, G., van Engelen, B., Vissing, J. & Raz, V., 1 Oct 2016, In : Human Molecular Genetics. 25, 19, p. 4282–4287 6 p.

    Research output: Contribution to journalArticle

  25. Published

    Advances in gene therapy for muscular dystrophies

    Abdul-Razak, H., Malerba, A. & Dickson, G., 18 Aug 2016, In : F1000Research. 5, p. 1-7 7 p.

    Research output: Contribution to journalArticle

  26. Published

    Strain-dependent and distinctive T-cell responses to HIV antigens following immunisation of mice with differing chimpanzee adenovirus vaccine vectors

    Herath, S., Le Heron, A., Colloca, S., Patterson, S., Tatoud, R., Weber, J. & Dickson, G., 17 Aug 2016, In : Vaccine. 34, 37, p. 4378-4385 8 p.

    Research output: Contribution to journalArticle

  27. Published

    Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

    Peccate, C., Mollard, A., Le Hir, M., Julien, L., McClorey, G., Jarmin, S., Le Heron, A., Dickson, G., Benkhelifa-Ziyyat, S., Piétri-Rouxel, F., Wood, M. J., Voit, T. & Lorain, S., 15 Aug 2016, In : Human Molecular Genetics. 25, 16, p. 3555–3563 9 p.

    Research output: Contribution to journalArticle

  28. Published
  29. Published

    piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

    Loperfido, M., Jarmin, S., Dastidar, S., Di Matteo, M., Perini, I., Moore, M., Nair, N., Samara-Kuko, E., Athanasopoulos, T., Tedesco, F. S., Dickson, G., Sampaolesi, M., VandenDriessche, T. & Chuah, M. K., 29 Jan 2016, In : Nucleic Acids Research. 44, 2, p. 744-760 17 p.

    Research output: Contribution to journalArticle

  30. E-pub ahead of print

    Antisense targeting of 3'end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for Facioscapulohumeral Dystrophy: a new gene silencing approach

    Marsollier, A-C., Ciszewski, L., Mariot, V., Popplewell, L., Voit, T., Dickson, G. & Dumonceaux, J., 19 Jan 2016, In : Human Molecular Genetics.

    Research output: Contribution to journalArticle

  31. 2015
  32. Published

    Analysis of T cell responses to chimpanzee adenovirus vectors encoding HIV gag-pol-nef antigen

    Herath, S., Le Heron, A., Colloca, S., Bergin, P., Patterson, S., Weber, J., Tatoud, R. & Dickson, G., 16 Dec 2015, In : Vaccine. 33, 51, p. 7283–7289 7 p.

    Research output: Contribution to journalArticle

  33. Published

    Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells

    Massouridès, E., Polentes, J., Mangeot, P-E., Mournetas, V., Nectoux, J., Deburgrave, N., Nusbaum, P., Leturcq, F., Popplewell, L., Dickson, G., Wein, N., Flanigan, K. M., Peschanski, M., Chelly, J. & Pinset, C., 14 Nov 2015, In : Skeletal Muscle. 5, 40, p. 1-18 18 p.

    Research output: Contribution to journalArticle

  34. Published

    Combination antisense treatment for destructive exon skipping of myostatin and open reading frame rescue of dystrophin in neonatal mdx mice

    Lu-Nguyen, N., Jarmin, S. A., Saleh, A. F., Popplewell, L., Gait, M. J. & Dickson, G., Aug 2015, In : Molecular Therapy. 23, 8, p. 1341-1348 8 p.

    Research output: Contribution to journalArticle

  35. Published

    Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy

    Rau, F., Lainé, J., Ramanoudjame, L., Ferry, A., Arandel, L., Delalande, O., Jollet, A., Dingli, F., Lee, K-Y., Peccate, C., Lorain, S., Kabashi, E., Athanasopoulos, T., Koo, T., Loew, D., Swanson, M. S., Le Rumeur, E., Dickson, G., Allamand, V., Marie, J. & 1 others, Furling, D., 28 May 2015, In : Nature Communications. 6, p. 1-10 10 p., 7205.

    Research output: Contribution to journalArticle

  36. Published

    Progress on Gene Therapy, Cell Therapy, and Pharmacological Strategies Toward the Treatment of Oculopharyngeal Muscular Dystrophy

    Harish, P., Malerba, A., Dickson, G. & Bachtarzi, H., 1 May 2015, In : Human Gene Therapy. 26, 5, p. 286-292 7 p.

    Research output: Contribution to journalArticle

  37. Published

    Mitochondrial Dysfunction Reveals the Role of mRNA Poly(A) Tail Regulation in Oculopharyngeal Muscular Dystrophy Pathogenesis

    Chartier, A., Klein, P., Pierson, S., Barbezier, N., Gidaro, T., Casas, F., Carberry, S., Dowling, P., Maynadier, L., Bellec, M., Oloko, M., Jardel, C., Moritz, B., Dickson, G., Mouly, V., Ohlendieck, K., Butler-Browne, G., Trollet, C. & Simonelig, M., 27 Mar 2015, In : PLoS genetics. 11, 3, p. 1-27 27 p., e1005092.

    Research output: Contribution to journalArticle

  38. Published

    Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

    Kawecka, K., Theodoulides, M., Hasoglu, Y., Jarmin, S., Kymalainen, H., Le-Heron, A., Popplewell, L., Malerba, A., Dickson, G. & Athanasopoulos, T., 2015, In : Current Gene Therapy. 15, 4, p. 395-415 21 p.

    Research output: Contribution to journalArticle

  39. 2014
  40. Published

    Genome-Wide Computational Analysis Reveals Cardiomyocyte-Specific Transcriptional CIS-Regulatory Motifs that Enable Efficient Cardiac Gene Therapy

    Rincon, M. Y., Sarcar, S., Danso-Abeam, D., Keyaerts, M., Matrai, J., Samara-Kuko, E., Acosta-Sanchez, A., Athanasopoulos, T., Dickson, G., Lahoutte, T., De Bleser, P., VandenDriessche, T. & Chuah, M. K., 8 Sep 2014, In : Molecular Therapy.

    Research output: Contribution to journalArticle

  41. Published

    Local Overexpression of the Myostatin Propeptide Increases Glucose Transporter Expression and Enhances Skeletal Muscle Glucose Disposal

    Cleasby, M. E., Jarmin, S., Eilers, W., Elashry, M., Andersen, D. K., Dickson, G. & Foster, K., 1 Apr 2014, In : American Journal of Physiology. Endocrinology and Metabolism.

    Research output: Contribution to journalArticle

  42. Published

    Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors (IDLVs)

    Kymalainen, H., Appelt, J-U., Giordano, F. A., Davies, A. F., Ogilvie, C. M., Ahmed, S. G., Laufs, S., Schmidt, M., Bode, J., Yáñez-Muñoz, R. J. & Dickson, G., 2 Feb 2014, In : Human Gene Therapy.

    Research output: Contribution to journalArticle

  43. Published

    New developments in the use of gene therapy to treat Duchenne muscular dystrophy

    Jarmin, S., Kymalainen, H., Popplewell, L. & Dickson, G., Feb 2014, In : Expert Opinion on Biological Therapy. 14, 2, p. 209-30 22 p.

    Research output: Contribution to journalArticle

  44. Published
  45. Published
  46. Published
  47. Published

    Fusion of Ubiquitin to HIV Gag Impairs Human Monocyte-Derived Dendritic Cell Maturation and Reduces Ability to Induce Gag T Cell Responses

    Herath, S., Benlahrech, A., Papagatsias, T., Athanasopoulos, T., Bouzeboudjen, Z., Hervouet, C., Klavinskis, L., Meiser, A., Kelleher, P., Dickson, G. & Patterson, S., 2014, In : PLoS ONE. 9, 2, p. e88327

    Research output: Contribution to journalArticle

  48. 2013
  49. Published

    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 (or Yáñez-Muñoz), R. J. & Dickson, G., Jul 2013, In : Human Gene Therapy. 24, 7, p. 692-701 10 p.

    Research output: Contribution to journalArticle

  50. Published

    TALEN-directed genome surgery of the DMD gene

    Popplewell, L., Marsh, J., Kymalainen, H., Yanez, R. & Dickson, J., 15 May 2013, Human Gene Therapy. Mary Ann Liebert Inc., Vol. 24. p. A21 1 p.

    Research output: Chapter in Book/Report/Conference proceedingConference contribution

  51. Published

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

    Tremblay, J. P., Xiao, X., Aartsma-Rus, A., Barbas, C., Blau, H. M., Bogdanove, A. J., Boycott, K., Braun, S., Breakefield, X. O., Bueren, J. A., Buschmann, M., Byrne, B. J., Calos, M., Cathomen, T., Chamberlain, J., Chuah, M., Cornetta, K., Davies, K. E., Dickson, J. G., Duchateau, P. & 31 others, Flotte, T. R., Gaudet, D., Gersbach, C. A., Gilbert, R., Glorioso, J., Herzog, R. W., High, K. A., Huang, W., Huard, J., Joung, J. K., Liu, D., Liu, D., Lochmüller, H., Lustig, L., Martens, J., Massie, B., Mavilio, F., Mendell, J. R., Nathwani, A., Ponder, K., Porteus, M., Puymirat, J., Samulski, J., Takeda, S., Thrasher, A., VandenDriessche, T., Wei, Y., Wilson, J. M., Wilton, S. D., Wolfe, J. H. & Gao, G., Feb 2013, In : Molecular Therapy. 21, 2, p. 266-8 3 p.

    Research output: Contribution to journalArticle

  52. Published

    Langerin negative dendritic cells promote potent CD8+ T-cell priming by skin delivery of live adenovirus vaccine microneedle arrays

    Bachy, V., Hervouet, C., Becker, P. D., Chorro, L., Carlin, L. M., Herath, S., Papagatsias, T., Barbaroux, J-B., Oh, S-J., Benlahrech, A., Athanasopoulos, T., Dickson, G., Patterson, S., Kwon, S-Y., Geissmann, F. & Klavinskis, L. S., 2013, In : Proceedings of the National Academy of Sciences of the United States of America.

    Research output: Contribution to journalArticle

  53. Published

    Nuclear entrapment and extracellular depletion of PCOLCE is associated with muscle degeneration in oculopharyngeal muscular dystrophy

    Raz, V., Sterrenburg, E., Routledge, S., Venema, A., van der Sluijs, B. M., Trollet, C., Dickson, G., van Engelen, B. G. M., van der Maarel, S. M. & Antoniou, M. N., 2013, In : BMC Neurology. 13, p. 70

    Research output: Contribution to journalArticle

  54. 2012
  55. Published

    Fragmentation of SIV-gag Vaccine Induces Broader T Cell Responses

    Benlahrech, A., Meiser, A., Herath, S., Papagatsias, T., Athanasopoulos, T., Li, F., Self, S., Bachy, V., Hervouet, C., Logan, K., Klavinskis, L., Dickson, G. & Patterson, S., 31 Oct 2012, In : PLoS ONE. 7, 10, 13 p., ARTN e48038.

    Research output: Contribution to journalArticle

  56. Published

    Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer

    Vandermeulen, G., Athanasopoulos, T., Trundley, A., Foster, K., Préat, V., Yáñez-Muñoz, R. J. & Dickson, G., 10 May 2012, In : Journal of Controlled Release. 159, 3, p. 376–383

    Research output: Contribution to journalArticle

  57. Published

    Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

    Daboussi, F., Zaslavskiy, M., Poirot, L., Loperfido, M., Gouble, A., Guyot, V., Leduc, S., Galetto, R., Grizot, S., Oficjalska, D., Perez, C., Delacôte, F., Dupuy, A., Chion-Sotinel, I., Le Clerre, D., Lebuhotel, C., Danos, O., Lemaire, F., Oussedik, K., Cédrone, F. & 11 others, Epinat, J-C., Smith, J., Yáñez (or Yáñez-Muñoz), R. J., Dickson, G., Popplewell, L., Koo, T., Vandendriessche, T., Chuah, M. K., Duclert, A., Duchateau, P. & Pâques, F., 2012, In : Nucleic Acids Research.

    Research output: Contribution to journalArticle

  58. Published

    Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

    Malerba, A., Kang, J. K., McClorey, G., Saleh, A. F., Popplewell, L., Gait, M. J., Wood, M. J. & Dickson, G., 2012, In : Molecular therapy. Nucleic acids. 1, p. e62

    Research output: Contribution to journalArticle

  59. Published

    Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy

    Foster, H., Popplewell, L. J. & Dickson, G., 2012, In : Human Gene Therapy.

    Research output: Contribution to journalLiterature review

  60. Published

    Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in duchenne muscular dystrophy

    Popplewell, L. J., Abu-Dayya, A., Khana, T., Flinterman, M., Khalique, N. A., Raju, L., Opstad, C. L., Sliwka, H-R., Partali, V., Dickson, G. & Pungente, M. D., 2012, In : Molecules. 17, 2, p. 1138-48 11 p.

    Research output: Contribution to journalArticle

  61. Published

    Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers

    Popplewell, L. J., Malerba, A. & Dickson, G., 2012, In : Methods in Molecular Biology. 867, p. 143-67 25 p.

    Research output: Contribution to journalArticle

  62. Published

    Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation

    de Klerk, E., Venema, A., Anvar, S. Y., Goeman, J. J., Hu, O., Trollet, C., Dickson, G., den Dunnen, J. T., van der Maarel, S. M., Raz, V. & 't Hoen, P. A. C., 2012, In : Nucleic Acids Research.

    Research output: Contribution to journalArticle

  63. 2011
  64. Published

    Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog

    Koo, T., Okada, T., Athanasopoulos, T., Foster, H., Takeda, S. & Dickson, G., Sep 2011, In : Journal of Gene Medicine. 13, 9, p. 497-506 10 p.

    Research output: Contribution to journalArticle

  65. Published

    Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

    Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M. E., Bourke, J., Wells, D. J., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Kole, R., Shrewsbury, S. B., Sewry, C., Morgan, J. E., Bushby, K. & Muntoni, F., 13 Aug 2011, In : The Lancet. 378, 9791, p. 595-605 11 p.

    Research output: Contribution to journalArticle

  66. Published

    Getting to the Heart of Things in the Field of Cardiovascular Gene Therapy

    Dickson, G., Aug 2011, In : Human Gene Therapy. 22, 8, p. 911-912 2 p.

    Research output: Contribution to journalEditorial

  67. Published
  68. Published

    Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

    Malerba, A., Sharp, P. S., Graham, I. R., Arechavala-Gomeza, V., Foster, K., Muntoni, F., Wells, D. J. & Dickson, G., Feb 2011, In : Molecular Therapy. 19, 2, p. 345-354

    Research output: Contribution to journalArticle

  69. Published
  70. Published

    Adeno-associated virus serotypes 7 and 8 outperform serotype 9 in expressing atheroprotective human apoE3 from mouse skeletal muscle

    Evans, V. C., Graham, I. R., Athanasopoulos, T., Galley, D. J., Jackson, C. L., Simons, J. P., Dickson, G. & Owen, J. S., 2011, In : Metabolism: clinical and experimental. 60, 4, p. 491-8

    Research output: Contribution to journalArticle

  71. Published
  72. Published

    Bioinformatic and Functional Optimization of Antisense Phosphorodiamidate Morpholino Oligomers (PMOs) for Therapeutic Modulation of RNA Splicing in Muscle

    Popplewell, L. J., Graham, I. R., Malerba, A. & Dickson, G., 2011, In : Methods in Molecular Biology. 709, p. 153-78 26 p.

    Research output: Contribution to journalArticle

  73. Published

    Codon optimization of the microdystrophin gene for duchene muscular dystrophy gene therapy

    Athanasopoulos, T., Foster, H., Foster, K. & Dickson, G., 2011, In : Methods in Molecular Biology. 709, p. 21-37 17 p.

    Research output: Contribution to journalArticle

  74. Published

    Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

    Pichavant, C., Aartsma-Rus, A., Clemens, P. R., Davies, K. E., Dickson, G., Takeda, S., Wilton, S. D., Wolff, J. A., Wooddell, C. I., Xiao, X. & Tremblay, J. P., 2011, In : Molecular Therapy. 19, 5, p. 830-40 11 p.

    Research output: Contribution to journalLiterature review

  75. Published
  76. Published

    Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients

    Anvar, S. Y., 't Hoen, P. A., Venema, A., van der Sluijs, B., van Engelen, B., Snoeck, M., Vissing, J., Trollet, C., Dickson, G., Chartier, A., Simonelig, M., van Ommen, G-J. B., van der Maarel, S. M. & Raz, V., 2011, In : Skeletal Muscle. 1, 1, p. 15

    Research output: Contribution to journalArticle

  77. Published

    Transcription Factor Rational Design Improves Directed Differentiation of Human Mesenchymal Stem Cells Into Skeletal Myocytes

    Gonçalves, M. A., Janssen, J. M., Nguyen, Q. G., Athanasopoulos, T., Hauschka, S. D., Dickson, G. & de Vries, A. A., 2011, In : Molecular Therapy. 19, 7, p. 1331-41

    Research output: Contribution to journalArticle

  78. 2010
  79. Published

    Canine microdystrophin results in remarkable expression and improvement in muscle pathology in a canine model of Duchenne Muscular Dystrophy following AAV gene transfer

    Koo, T., Shin, J. H., Athanasopoulos, T., Takashi, O., Foster, H., Shin'ichi, T. & Dickson, G., Oct 2010, In : Human Gene Therapy. 21, 10, p. 1469-1470 2 p.

    Research output: Contribution to journalArticle

  80. Published

    Evaluation of the truncated products of exon and multiple exon skipping in DMD therapy

    Foster, K., Jarmin, S., Beley, C., Foster, H., Garcia, L. & Dickson, G., Oct 2010, In : Neuromuscular disorders. 20, 9-10, p. 642 1 p.

    Research output: Contribution to journalArticle

  81. Published

    Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres

    Trollet, C., Anvar, S. Y., Venema, A., Hargreaves, I. P., Foster, K., Vignaud, A., Ferry, A., Negroni, E., Hourde, C., Baraibar, M. A., 't Hoen, P. A. C., Davies, J. E., Rubinsztein, D. C., Heales, S. J., Mouly, V., van der Maarel, S. M., Butler-Browne, G., Raz, V. & Dickson, G., 1 Jun 2010, In : Human Molecular Genetics. 19, 11, p. 2191-2207 17 p.

    Research output: Contribution to journalArticle

  82. Published

    Antisense Oligonucleotide-Induced Multiexon Skipping in Duchenne Muscular Dystrophy

    Popplewell, L. J. & Dickson, G., Apr 2010, In : Human Gene Therapy. 21, 4, p. 507-508 2 p.

    Research output: Contribution to journalMeeting abstract

  83. Published

    Challenges with advanced therapy medicinal products and how to meet them

    Schneider, C. K., Salmikangas, P., Jilma, B., Flamion, B., Todorova, L. R., Paphitou, A., Haunerova, I., Maimets, T., Trouvin, J-H., Flory, E., Tsiftsoglou, A., Sarkadi, B., Gudmundsson, K., O'Donovan, M., Migliaccio, G., Ancāns, J., Maciulaitis, R., Robert, J-L., Samuel, A., Ovelgönne, J. H. & 31 others, Hystad, M., Fal, A. M., Lima, B. S., Moraru, A. S., Turcáni, P., Zorec, R., Ruiz, S., Akerblom, L., Narayanan, G., Kent, A., Bignami, F., Dickson, J. G., Niederwieser, D., Figuerola-Santos, M-A., Reischl, I. G., Beuneu, C., Georgiev, R., Vassiliou, M., Pychova, A., Clausen, M., Methuen, T., Lucas, S., Schüssler-Lenz, M., Kokkas, V., Buzás, Z., MacAleenan, N., Galli, M. C., Linē, A., Gulbinovic, J., Berchem, G. & Committee for Advanced Therapies (CAT), 2010, In : Nature Reviews. Drug Discovery. 9, 3, p. 195-201 7 p.

    Research output: Contribution to journalArticle

  84. Published

    Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

    Popplewell, L. J., Adkin, C., Arechavala-Gomeza, V., Aartsma-Rus, A., de Winter, C. L., Wilton, S. D., Morgan, J. E., Muntoni, F., Graham, I. R. & Dickson, G., 2010, In : Neuromuscular disorders. 20, 2, p. 102-10 9 p.

    Research output: Contribution to journalArticle

  85. Published

    Production of non viral DNA vectors

    Schleef, M., Blaesen, M., Schmeer, M., Baier, R., Marie, C., Dickson, G. & Scherman, D., 2010, In : Current Gene Therapy. 10, 6, p. 487-507 21 p.

    Research output: Contribution to journalArticle

  86. Published

    Transcriptomic analysis of dystrophin RNAi knockdown reveals a central role for dystrophin in muscle differentiation and contractile apparatus organization

    Ghahramani Seno, M. M., Trollet, C., Athanasopoulos, T., Graham, I. R., Hu, P. & Dickson, G., 2010, In : BMC Genomics. 11, p. 345

    Research output: Contribution to journalArticle

  87. 2009
  88. Published

    Development of thermo-stable Ad-based vaccines expressing ubiquitin fusions of full or fragmented HIV/SIV genetic components

    Athanasopoulos, T., Papagatsias, T., Meiser, A., Harris, J., Bachy, V., Benlahrech, A., Herath, S., Roesen, N., Oh, S., Li, F., Self, S., Kwon, S-Y., Fischer, K., Seymour, L., Daniels, R., Almond, N., Klavinskis, L., Logan, K., Patterson, S. & Dickson, G., Nov 2009, In : Human Gene Therapy. 20, 11, p. 1392-1393 2 p.

    Research output: Contribution to journalArticle

  89. Published

    Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

    Kinali, M., Arechavala-Gomeza, V., Feng, L., Cirak, S., Hunt, D., Adkin, C., Guglieri, M., Ashton, E., Abbs, S., Nihoyannopoulos, P., Garralda, M. E., Rutherford, M., McCulley, C., Popplewell, L., Graham, I. R., Dickson, G., Wood, M. J. A., Wells, D. J., Wilton, S. D., Kole, R. & 5 others, Straub, V., Bushby, K., Sewry, C., Morgan, J. E. & Muntoni, F., Oct 2009, In : Lancet Neurology. 8, 10, p. 918-928 11 p.

    Research output: Contribution to journalArticle

  90. Published

    Gene therapy for muscular dystrophy: current progress and future prospects

    Trollet, C., Athanasopoulos, T., Popplewell, L., Malerba, A. & Dickson, G., Jul 2009, In : Expert Opinion on Biological Therapy. 9, 7, p. 849-866 18 p.

    Research output: Contribution to journalLiterature review

  91. Published

    Adeno-associated virus-8-mediated intravenous transfer of myostatin propeptide leads to systemic functional improvements of slow but not fast muscle

    Foster, K., Graham, I. R., Otto, A., Foster, H., Trollet, C., Yaworsky, P. J., Walsh, F. S., Bickham, D., Curtin, N. A., Kawar, S. L., Patel, K. & Dickson, G., 2009, In : Rejuvenation Research. 12, 2, p. 85-94 10 p.

    Research output: Contribution to journalArticle

  92. Published

    Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1

    Benlahrech, A., Harris, J., Meiser, A., Papagatsias, T., Hornig, J., Hayes, P., Lieber, A., Athanasopoulos, T., Bachy, V., Csomor, E., Daniels, R., Fisher, K., Gotch, F., Seymour, L., Logan, K., Barbagallo, R., Klavinskis, L., Dickson, G. & Patterson, S., 2009, In : Proceedings of the National Academy of Sciences of the United States of America. 106, 47, p. 19940-5 6 p.

    Research output: Contribution to journalArticle

  93. Published

    Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene

    Popplewell, L. J., Trollet, C., Dickson, G. & Graham, I. R., 2009, In : Molecular Therapy. 17, 3, p. 554-61 8 p.

    Research output: Contribution to journalArticle

  94. Published

    Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice

    Malerba, A., Thorogood, F. C., Dickson, G. & Graham, I. R., 2009, In : Human Gene Therapy. 20, 9, p. 955-65 11 p.

    Research output: Contribution to journalArticle

  95. Published

    Efficient liver-directed gene transfer by in situ generation of retroviral vector from adenoviral templates

    Harris, J. D., Roberts, M. L., Perumal, D., Graham, I. R., Patterson, S. & Dickson, G., 2009, In : In Vivo. 23, 6, p. 885-93 9 p.

    Research output: Contribution to journalArticle

  96. Published

    Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice

    Foster, K., Matsakas, A., Otto, A., Macharia, R., Elashry, M. I., Feist, S., Graham, I., Foster, H., Yaworsky, P., Walsh, F., Dickson, G. & Patel, K., 2009, In : Neuromuscular disorders. 19, 7, p. 489-99 11 p.

    Research output: Contribution to journalArticle

  97. Published

    Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice

    Osman, E., Evans, V., Graham, I. R., Athanasopoulos, T., McIntosh, J., Nathwani, A. C., Simons, J. P., Dickson, G. & Owen, J. S., 2009, In : Atherosclerosis. 204, 1, p. 121-6 6 p.

    Research output: Contribution to journalArticle

  98. 2008
  99. Published
  100. Published

    Activity of different vaccine-associated promoter elements in human dendritic cells

    Papagatsias, T., Rozis, G., Athanasopoulos, T., Gotch, F., Dickson, G. & Patterson, S., 2008, In : Immunology Letters. 115, 2, p. 117-25 9 p.

    Research output: Contribution to journalArticle

  101. Published

    Adenovirus-based targeting in myoblasts is hampered by nonhomologous vector integration

    Isman, O., Roberts, M. L., Morgan, J. E., Graham, I. R., Goldring, K., Lawrence-Watt, D. J., Lu, Q. L., Dunckley, M. G., Porter, A. C. G., Partridge, T. A. & Dickson, G., 2008, In : Human Gene Therapy. 19, 10, p. 1000-8 9 p.

    Research output: Contribution to journalArticle

  102. Published

    Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer

    Foster, H., Sharp, P. S., Athanasopoulos, T., Trollet, C., Graham, I. R., Foster, K., Wells, D. J. & Dickson, G., 2008, In : Molecular Therapy. 16, 11, p. 1825-32 8 p.

    Research output: Contribution to journalArticle

  103. Published

    Human apolipoprotein E expression from mouse skeletal muscle by electrotransfer of nonviral DNA (plasmid) and pseudotyped recombinant adeno-associated virus (AAV2/7)

    Evans, V., Foster, H., Graham, I. R., Foster, K., Athanasopoulos, T., Simons, J. P., Dickson, G. & Owen, J. S., 2008, In : Human Gene Therapy. 19, 6, p. 569-78 10 p.

    Research output: Contribution to journalArticle

  104. Published

    RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology

    Ghahramani Seno, M. M., Graham, I. R., Athanasopoulos, T., Trollet, C., Pohlschmidt, M., Crompton, M. R. & Dickson, G., 2008, In : Human Molecular Genetics. 17, 17, p. 2622-32 11 p.

    Research output: Contribution to journalArticle

  105. 2007
  106. Published

    Comparative analysis of antisense oligonucleotide sequences for targeted skipping of Exon 51 during dystrophin Pre-mRNA splicing in human muscle

    Arechavala-Gomeza, V., Graham, I. R., Popplewell, L. J., Adams, A. M., Aartsma-Rus, A., Kinali, M., Morgan, J. E., Van Deutekom, J. C., Wilton, S. D., Dickson, G. & Muntoni, F., Sep 2007, In : Human Gene Therapy. 18, 9, p. 798-810 13 p.

    Research output: Contribution to journalArticle

  107. Published

    APOE epsilon3 gene transfer attenuates brain damage after experimental stroke

    McColl, B. W., McGregor, A. L., Wong, A., Harris, J. D., Amalfitano, A., Magnoni, S., Baker, A. H., Dickson, G. & Horsburgh, K., 2007, In : Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism. 27, 3, p. 477-87 11 p.

    Research output: Contribution to journalArticle

  108. 2006
  109. Published

    Delivery of human apolipoprotein (apo) E to liver by an [E1(-), E3(-), polymerase(-), pTP(-)] adenovirus vector containing a liver-specific promoter inhibits atherogenesis in immunocompetent apoE-deficient mice

    Harris, J. D., Graham, I. R., Amalfitano, A., Owen, J. S. & Dickson, G., 2006, In : GENE THERAPY AND MOLECULAR BIOLOGY. 10A, p. 17-29 13 p.

    Research output: Contribution to journalArticle

  110. Published

    Gene therapy progress and prospects: Duchenne muscular dystrophy

    Foster, K., Foster, H. & Dickson, J. G., 2006, In : Human Gene Therapy. 13, 24, p. 1677-85 9 p.

    Research output: Contribution to journalArticle

  111. Published

    Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice

    Ito, K., Kimura, S., Ozasa, S., Matsukura, M., Ikezawa, M., Yoshioka, K., Ueno, H., Suzuki, M., Araki, K., Yamamura, K., Miwa, T., Dickson, G., Thomas, G. D. & Miike, T., 2006, In : Human Molecular Genetics. 15, 14, p. 2266-75 10 p.

    Research output: Contribution to journalArticle

  112. 2005
  113. Published

    Apolipoprotein E delivery by peritoneal implantation of encapsulated recombinant cells improves the hyperlipidaemic profile in apoE-deficient mice

    Tagalakis, A. D., Diakonov, I. A., Graham, I. R., Heald, K. A., Harris, J. D., Mulcahy, J. V., Dickson, G. & Owen, J. S., 2005, In : BBA - Gene Structure and Expression . 1686 3, p. 190-9 10 p.

    Research output: Contribution to journalArticle

  114. Published

    Correction of the neuropathogenic human apolipoprotein E4 (APOE4) gene to APOE3 in vitro using synthetic RNA/DNA oligonucleotides (chimeraplasts)

    Tagalakis, A. D., Dickson, J. G., Owen, J. S. & Simons, J. P., 2005, In : Journal of molecular neuroscience : MN. 25, 1, p. 95-103 9 p.

    Research output: Contribution to journalArticle

  115. Published

    Langerhans cells are more efficiently transduced than dermal dendritic cells by adenovirus vectors expressing either group C or group B fibre protein: implications for mucosal vaccines

    Rozis, G., de Silva, S., Benlahrech, A., Papagatsias, T., Harris, J., Gotch, F., Dickson, G. & Patterson, S., 2005, In : European Journal of Immunology. 35, 9, p. 2617-26 10 p.

    Research output: Contribution to journalArticle

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