Dr Ngoc Lu-Nguyen

  1. 2020
  2. Published

    G-quadruplex ligands mediate downregulation of DUX4 expression

    Ciszewski, L., Lu-Nguyen, N., Dickson, G. & Popplewell, L., 17 Mar 2020, In : Nucleic Acids Research. p. 1-16 16 p., gkaa146.

    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

    Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown

    Golshirazi, G., Ciszewski, L., Lu-Nguyen, N. & Popplewell, L., 1 Sep 2018, In : Methods in Molecular Biology. 1828, p. 91-124 34 p.

    Research output: Contribution to journalArticle

  11. 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

  12. Published

    Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides

    March, J. T., Golshirazi, G., Cernisova, V., Carr, H., Leong, Y., Lu-Nguyen, N. & Popplewell, L. J., 25 Jun 2018, In : Biomedicines. 6, 3

    Research output: Contribution to journalReview article

  13. 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

  14. 2017
  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. E-pub ahead of print

    Intrastriatal Delivery of Integration-Deficient Lentiviral Vectors in a Rat Model of Parkinson’s Disease

    Lu-Nguyen, N., Broadstock, M. & Yáñez-Muñoz, R. J., 18 Jun 2016, In : Methods in Molecular Biology. 1448, p. 175-184 10 p.

    Research output: Contribution to journalArticle

  18. 2015
  19. Published
  20. 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

  21. 2014
  22. Published

    Transgenic expression of human glial cell line-derived neurotrophic factor (hGDNF) from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease

    Lu-Nguyen, N., Broadstock, M., Schliesser, M. G., Bartholomae, C., von Kalle, C., Schmidt, M. & Yáñez-Muñoz, R. J., 18 Mar 2014, In : Human Gene Therapy.

    Research output: Contribution to journalArticle

  23. 2013
  24. Unpublished