Combination Antisense Treatment for Duchenne Muscular Dystrophy: Open Reading Frame Rescue of Dystrophin in conjunction with Destructive Exon Skipping of Myostatin mRNA

Research output

Research output per year 2015 2015 2017 2018 2020 2020

• 4 Article
• 1 Review article

Research output per year

Research output per year

Search results

• 2020

  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., Jun 2020, In: Journal of Cachexia, Sarcopenia and Muscle. 11, 3, p. 768-782 15 p.

  Research output: Contribution to journal › Article › peer-review

  Open Access
• 2018

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

  Lu-Nguyen, N., Dickson, G. & Malerba, A., 1 Sept 2018, (E-pub ahead of print) In: Methods in Molecular Biology. 1828, p. 343-354 12 p.

  Research output: Contribution to journal › Article › peer-review
• 2017

  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 journal › Article › peer-review

  Open Access
• 2015

  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 journal › Article › peer-review

  Open Access

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