Dr Alberto Malerba

  1. 2020
  2. Published

    Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease

    Harish, P., Forrest, L., Herath, S., Dickson, G., Malerba, A. & Popplewell, L., 5 Mar 2020, In : Frontiers in Physiology. 11, p. 1-8 8 p., 184.

    Research output: Contribution to journalArticle

  3. E-pub ahead of print

    Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

    Zhou, H., Meng, J., Malerba, A., Catapano, F., Sintusek, P., Jarmin, S., Feng, L., Lu-Nguyen, N., Sun, L., Mariot, V., Dumonceaux, J., Morgan, J. E., Gissen, P., Dickson, G. & Muntoni, F., 7 Feb 2020, In : Journal of Cachexia, Sarcopenia and Muscle. p. 1-15 15 p.

    Research output: Contribution to journalArticle

  4. 2019
  5. Published

    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., 1 Oct 2019, In : Human Molecular Genetics. p. 1-24 24 p., ddz167.

    Research output: Contribution to journalArticle

  6. 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

  7. 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

  8. 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

  9. 2018
  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. 2017
  13. 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

  14. 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

  15. 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

  16. 2016
  17. 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

  18. 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

  19. 2015
  20. 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

  21. 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

  22. 2014
  23. Published
  24. 2012
  25. 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

  26. 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

  27. 2011
  28. Published
  29. 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

  30. Published
  31. Published
  32. 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

  33. Published
  34. 2009
  35. 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

  36. 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