Destructive Exon Skipping of Myostatin mRNA with and without Open Reading Frame Rescue of Dystrophin

Project: Research

Status	Finished
Effective start/end date	1/01/14 → 30/06/15

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
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., 15 Sept 2019, In: Human Molecular Genetics. 28, 18, p. 3091-3100 10 p., ddz125.
Research output: Contribution to journal › Article › peer-review

Open Access
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50 Downloads (Pure)
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

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

Project Details