Dr Alberto Malerba

  1. 2021
  2. Published

    BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

    Strings-Ufombah, V., Malerba, A., Kao, S-C., Harbaran, S., Roth, F., Cappellari, O., Lu-Nguyen, N., Takahashi, K., Mukadam, S., Kilfoil, G., Kloth, C., Roelvink, P., Dickson, G., Trollet, C. & Suhy, D., 4 Jun 2021, In: Molecular therapy. Nucleic acids. 24, p. 67-78 12 p.

    Research output: Contribution to journalArticlepeer-review

  3. E-pub ahead of print

    Knockdown of Muscle-Specific Ribosomal Protein L3-Like Enhances Muscle Function in Healthy and Dystrophic Mice

    Kao, B. R., Malerba, A., Lu-Nguyen, N. B., Harish, P., McCarthy, J. J., Dickson, G. & Popplewell, L. J., 2 Jun 2021, (E-pub ahead of print) In: Nucleic Acid Therapeutics.

    Research output: Contribution to journalArticlepeer-review

  4. E-pub ahead of print

    Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model

    Lu-Nguyen, N., Malerba, A., Herath, S., Dickson, G. & Popplewell, L., 13 May 2021, (E-pub ahead of print) In: Human Molecular Genetics. ddab136.

    Research output: Contribution to journalArticlepeer-review

  5. E-pub ahead of print

    Dose-dependent microdystrophin expression enhancement in cardiac muscle by a cardiac specific regulatory element

    Malerba, A., Sidoli, C., Lu-Nguyen, N. B., Herath, S., Le Heron, A., Abdul-Razak, H., Jarmin, S., Vandendriessche, T., Chuah, M. K. L., Dickson, G. & Popplewell, L. J., 26 Mar 2021, (E-pub ahead of print) In: Human Gene Therapy.

    Research output: Contribution to journalArticlepeer-review

  6. 2020
  7. 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 journalArticlepeer-review

  8. 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, (E-pub ahead of print) In: Journal of Cachexia, Sarcopenia and Muscle. p. 1-15 15 p.

    Research output: Contribution to journalArticlepeer-review

  9. 2019
  10. 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 journalArticlepeer-review

  11. Published

    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, G., Oct 2019, In: Journal of Cachexia, Sarcopenia and Muscle. 10, 5, p. 1016-1026 11 p.

    Research output: Contribution to journalArticlepeer-review

  12. 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, (E-pub ahead of print) In: Human Molecular Genetics. ddz125.

    Research output: Contribution to journalArticlepeer-review

  13. 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 journalArticlepeer-review

  14. 2018
  15. 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, (E-pub ahead of print) In: Methods in Molecular Biology. 1828, p. 343-354 12 p.

    Research output: Contribution to journalArticlepeer-review

  16. 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 journalArticlepeer-review

  17. 2017
  18. 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 journalArticlepeer-review

  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 journalArticlepeer-review

  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 journalArticlepeer-review

  21. 2016
  22. 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 journalArticlepeer-review

  23. 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 journalArticlepeer-review

  24. 2015
  25. 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 journalArticlepeer-review

  26. 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 journalArticlepeer-review

  27. 2014
  28. Published
  29. 2012
  30. 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 journalArticlepeer-review

  31. 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 journalArticlepeer-review

  32. 2011
  33. Published

    Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity

    Malerba, A., Boldrin, L. & Dickson, G., Aug 2011, In: Nucleic Acid Therapeutics. 21, 4, p. 293-298 6 p.

    Research output: Contribution to journalArticlepeer-review

  34. 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 journalArticlepeer-review

  35. Published

    Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa-guanidine Morpholino Oligomer Treatment

    Kang, J. K., Malerba, A., Popplewell, L., Foster, K. & Dickson, G., Jan 2011, In: Molecular Therapy. 19, 1, p. 159-164 6 p.

    Research output: Contribution to journalArticlepeer-review

  36. Published
  37. 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 journalArticlepeer-review

  38. Published
  39. 2009
  40. 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 reviewpeer-review

  41. 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 journalArticlepeer-review