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

Project: Research

Status	Finished
Effective start/end date	1/02/12 → 31/08/15

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
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
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 journal › Review article › peer-review

Networking towards clinical application of antisense-mediated exon skipping (Portugal)
Lu-Nguyen, N. (Participant)
2017
Activity: Participating in or organising an event › Participation in a conference
MDEX Consortium Meeting (UK)
Lu-Nguyen, N. (Speaker)
2016
Activity: Participating in or organising an event › Workshop, seminar, course or webinar.
Workshop on delivery and delivery to the central nervous system (Spain)
Lu-Nguyen, N. (Speaker)
2016
Activity: Talk, presentation or media contribution › Oral presentation

Fellowship - European Cooperation in Science and Technology
Lu-Nguyen, N. (Recipient), 2016
Prize: Prize (including medals and awards)
Fellowship - European Cooperation in Science and Technology
Lu-Nguyen, N. (Recipient), 2015
Prize: Prize (including medals and awards)
Travel Grant - British Society for Gene and Cell Therapy
Lu-Nguyen, N. (Recipient), 2016
Prize: Prize (including medals and awards)

Project Details