Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies. / Dickson, George.

2014. S3 Abstract from 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom.

Research output: Contribution to conferenceAbstract

Published

Standard

Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies. / Dickson, George.

2014. S3 Abstract from 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom.

Research output: Contribution to conferenceAbstract

Harvard

Dickson, G 2014, 'Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies', 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom, 3/03/14 - 4/03/14 pp. S3.

APA

Dickson, G. (2014). Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies. S3. Abstract from 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom.

Vancouver

Dickson G. Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies. 2014. Abstract from 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom.

Author

Dickson, George. / Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies. Abstract from 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders, London, United Kingdom.

BibTeX

@conference{d8a9684e1c5047eab1267ceba181c0ec,
title = "Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies",
abstract = "Myostatin, a secreted growth factor of the TGFb family, is expressedin skeletal muscle and adipose tissue. Myostatin expression limitsthe size of muscle during development, and myostatin mutantanimals exhibit dramatic increases in muscle mass, reduction in fatmass and resistance to diet-induced and genetic obesity. Inhibitionof the myostatin pathway represents a potential therapeutic targetto reverse muscle wasting in a range of disease situations suchas muscular dystrophies and atrophies, sarcopenia, and diseaseinduced(COPD, cancer, diabetes) muscle cachexia. Use of blockingantibodies, and of natural or engineered inhibitory binding partnersto myostatin such as myostatin propeptide, follistatin and solubleActRIIB receptor fragments has resulted in increase in muscle mass.These protein delivery strategies are expensive, require repeated dosing, lead to fluctuating levels of active circulating reagent,and can be accompanied by undesirable immune responses.Another method to deliver these therapeuics is via gene therapy,involving the use of non-viral or viral vectors to provide a longterm gene-based expression of myostatin inhibitors. Finally, thereare oligonucleotide-based strategies to knock-down myostain viaRNA interference or antisense mechanisms. Exon skipping is oneantisense strategy for knocking down targeted transcripts, byinterference with the natural pre-mRNA splicing pattern. We havedesigned, and evaluated the use of, antisense oligonucleotides of2O-methyl phosphorothioate and phosphodiamidate morpholinochemistry targeting the myostatin pre-mRNA to perturb its splicingand provide a mechanism to efficiently inhibit myostatin geneexpression activity.",
author = "George Dickson",
year = "2014",
language = "English",
pages = "S3",
note = "7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders ; Conference date: 03-03-2014 Through 04-03-2014",

}

RIS

TY - CONF

T1 - Antisense approaches to counter skeletal muscle atrophy and fibrosis: targeting myostatin and other strategies

AU - Dickson, George

PY - 2014

Y1 - 2014

N2 - Myostatin, a secreted growth factor of the TGFb family, is expressedin skeletal muscle and adipose tissue. Myostatin expression limitsthe size of muscle during development, and myostatin mutantanimals exhibit dramatic increases in muscle mass, reduction in fatmass and resistance to diet-induced and genetic obesity. Inhibitionof the myostatin pathway represents a potential therapeutic targetto reverse muscle wasting in a range of disease situations suchas muscular dystrophies and atrophies, sarcopenia, and diseaseinduced(COPD, cancer, diabetes) muscle cachexia. Use of blockingantibodies, and of natural or engineered inhibitory binding partnersto myostatin such as myostatin propeptide, follistatin and solubleActRIIB receptor fragments has resulted in increase in muscle mass.These protein delivery strategies are expensive, require repeated dosing, lead to fluctuating levels of active circulating reagent,and can be accompanied by undesirable immune responses.Another method to deliver these therapeuics is via gene therapy,involving the use of non-viral or viral vectors to provide a longterm gene-based expression of myostatin inhibitors. Finally, thereare oligonucleotide-based strategies to knock-down myostain viaRNA interference or antisense mechanisms. Exon skipping is oneantisense strategy for knocking down targeted transcripts, byinterference with the natural pre-mRNA splicing pattern. We havedesigned, and evaluated the use of, antisense oligonucleotides of2O-methyl phosphorothioate and phosphodiamidate morpholinochemistry targeting the myostatin pre-mRNA to perturb its splicingand provide a mechanism to efficiently inhibit myostatin geneexpression activity.

AB - Myostatin, a secreted growth factor of the TGFb family, is expressedin skeletal muscle and adipose tissue. Myostatin expression limitsthe size of muscle during development, and myostatin mutantanimals exhibit dramatic increases in muscle mass, reduction in fatmass and resistance to diet-induced and genetic obesity. Inhibitionof the myostatin pathway represents a potential therapeutic targetto reverse muscle wasting in a range of disease situations suchas muscular dystrophies and atrophies, sarcopenia, and diseaseinduced(COPD, cancer, diabetes) muscle cachexia. Use of blockingantibodies, and of natural or engineered inhibitory binding partnersto myostatin such as myostatin propeptide, follistatin and solubleActRIIB receptor fragments has resulted in increase in muscle mass.These protein delivery strategies are expensive, require repeated dosing, lead to fluctuating levels of active circulating reagent,and can be accompanied by undesirable immune responses.Another method to deliver these therapeuics is via gene therapy,involving the use of non-viral or viral vectors to provide a longterm gene-based expression of myostatin inhibitors. Finally, thereare oligonucleotide-based strategies to knock-down myostain viaRNA interference or antisense mechanisms. Exon skipping is oneantisense strategy for knocking down targeted transcripts, byinterference with the natural pre-mRNA splicing pattern. We havedesigned, and evaluated the use of, antisense oligonucleotides of2O-methyl phosphorothioate and phosphodiamidate morpholinochemistry targeting the myostatin pre-mRNA to perturb its splicingand provide a mechanism to efficiently inhibit myostatin geneexpression activity.

M3 - Abstract

SP - S3

T2 - 7th Annual UK Neuromuscular Translational Research Conference, 2014 /Neuromuscular Disorders

Y2 - 3 March 2014 through 4 March 2014

ER -