Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Ngoc Lu-Nguyen; Alberto Malerba; Linda Popplewell; Fred Schnell; Gunnar Hanson; George Dickson

doi:10.1016/j.omtn.2016.11.009

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell, Fred Schnell, Gunnar Hanson, George Dickson

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

Original language	English
Pages (from-to)	15-28
Number of pages	14
Journal	Molecular therapy. Nucleic acids
Volume	6
Early online date	14 Dec 2016
DOIs	https://doi.org/10.1016/j.omtn.2016.11.009
Publication status	Published - 17 Mar 2017

Keywords

Journal Article

Access to Document

10.1016/j.omtn.2016.11.009Licence: CC BY

Destructive Exon Skipping of Myostatin mRNA with and without Open Reading Frame Rescue of Dystrophin
Lu-Nguyen, N. (Researcher) & Dickson, G. (PI)
Industrial
1/01/14 → 30/06/15
Project: Research
Combination Antisense Treatment for Duchenne Muscular Dystrophy: Open Reading Frame Rescue of Dystrophin in conjunction with Destructive Exon Skipping of Myostatin mRNA
Lu-Nguyen, N. (Researcher) & Dickson, G. (PI)
Muscular Dystrophy Campaign
1/02/12 → 31/08/15
Project: Research

Workshop on delivery and delivery to the central nervous system (Spain)
Lu-Nguyen, N. (Speaker)
2016
Activity: Talk or presentation › Oral presentation
Hellenic Society of Gene Therapy and Regenerative Medicine Congress (Greece)
Lu-Nguyen, N. (Speaker)
2016
Activity: Talk or presentation › Oral presentation
Training school on patient communication (Netherlands)
Lu-Nguyen, N. (Speaker)
2015
Activity: Talk or presentation › Oral presentation

Travel Grant - British Society for Gene and Cell Therapy
Lu-Nguyen, Ngoc (Recipient), 2016
Prize: Prize (including medals and awards)

Cite this

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title = "Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice",

keywords = "Journal Article",

author = "Ngoc Lu-Nguyen and Alberto Malerba and Linda Popplewell and Fred Schnell and Gunnar Hanson and George Dickson",

year = "2017",

month = mar,

day = "17",

doi = "10.1016/j.omtn.2016.11.009",

language = "English",

volume = "6",

pages = "15--28",

journal = "Molecular therapy. Nucleic acids",

publisher = "Nature Publishing Group",

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T1 - Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

AU - Lu-Nguyen, Ngoc

AU - Malerba, Alberto

AU - Popplewell, Linda

AU - Schnell, Fred

AU - Hanson, Gunnar

AU - Dickson, George

PY - 2017/3/17

Y1 - 2017/3/17

KW - Journal Article

U2 - 10.1016/j.omtn.2016.11.009

DO - 10.1016/j.omtn.2016.11.009

M3 - Article

C2 - 28325281

VL - 6

SP - 15

EP - 28

JO - Molecular therapy. Nucleic acids

JF - Molecular therapy. Nucleic acids

ER -

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Keywords

Access to Document

Projects

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

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

Activities

Workshop on delivery and delivery to the central nervous system (Spain)

Hellenic Society of Gene Therapy and Regenerative Medicine Congress (Greece)

Training school on patient communication (Netherlands)

Prizes

Travel Grant - British Society for Gene and Cell Therapy

Cite this