Trends Mol Med. 2006 Mar;12(3):122-9. Epub 2006 Jan 27.

Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?

Source

Department of Cellular and Molecular Medicine and Centre for Neuromuscular Disease, Faculty of Medicine, University of Ottawa, 451 Smyth Road, Ottawa, Ontario K1H 8M5, Canada.

Abstract

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder for which there is currently no effective treatment. This disorder is caused by mutations or deletions in the gene encoding dystrophin that prevent expression of dystrophin at the sarcolemma. A promising pharmacological treatment for DMD aims to increase levels of utrophin, a homolog of dystrophin, in muscle fibers of affected patients to compensate for the absence of dystrophin. Here, we review recent developments in our understanding of the regulatory pathways that govern utrophin expression, and highlight studies that have used activators of these pathways to alleviate the dystrophic symptoms in DMD animal models. The results of these preclinical studies are promising and bring us closer to implementing appropriate utrophin-based drug therapies for DMD patients.

PMID:: 16443393; [PubMed - indexed for MEDLINE]

LinkOut - more resources

Full Text Sources

Supplemental Content

Save items

Related citations in PubMed

Review Utrophin upregulation in Duchenne muscular dystrophy. [Acta Myol. 2005]
Review Utrophin upregulation in Duchenne muscular dystrophy.
Hirst RC, McCullagh KJ, Davies KE. Acta Myol. 2005 Dec; 24(3):209-16.
Review Therapeutic strategies for Duchenne and Becker dystrophies. [Int Rev Cytol. 2004]
Review Therapeutic strategies for Duchenne and Becker dystrophies.
Voisin V, de la Porte S. Int Rev Cytol. 2004; 240:1-30.
Review Multiple regulatory events controlling the expression and localization of utrophin in skeletal muscle fibers: insights into a therapeutic strategy for Duchenne muscular dystrophy. [J Physiol Paris. 2002]
Review Multiple regulatory events controlling the expression and localization of utrophin in skeletal muscle fibers: insights into a therapeutic strategy for Duchenne muscular dystrophy.
Jasmin BJ, Angus LM, Bélanger G, Chakkalakal JV, Gramolini AO, Lunde JA, Stocksley MA, Thompson J. J Physiol Paris. 2002 Jan-Mar; 96(1-2):31-42.
Analysis of gene expression differences between utrophin/dystrophin-deficient vs mdx skeletal muscles reveals a specific upregulation of slow muscle genes in limb muscles. [Neurogenetics. 2006]
Analysis of gene expression differences between utrophin/dystrophin-deficient vs mdx skeletal muscles reveals a specific upregulation of slow muscle genes in limb muscles.
Baker PE, Kearney JA, Gong B, Merriam AP, Kuhn DE, Porter JD, Rafael-Fortney JA. Neurogenetics. 2006 May; 7(2):81-91. Epub 2006 Mar 9.
Nitric oxide and l-arginine cause an accumulation of utrophin at the sarcolemma: a possible compensation for dystrophin loss in Duchenne muscular dystrophy. [Neurobiol Dis. 1999]
Nitric oxide and l-arginine cause an accumulation of utrophin at the sarcolemma: a possible compensation for dystrophin loss in Duchenne muscular dystrophy.
Chaubourt E, Fossier P, Baux G, Leprince C, Israël M, De La Porte S. Neurobiol Dis. 1999 Dec; 6(6):499-507.

See reviews... See all...

Cited by 16 PubMed Central articles

Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. [PLoS One. 2011]
Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.
Tinsley JM, Fairclough RJ, Storer R, Wilkes FJ, Potter AC, Squire SE, Powell DS, Cozzoli A, Capogrosso RF, Lambert A, et al. PLoS One. 2011 May 6; 6(5):e19189. Epub 2011 May 6.
Review Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. [Mol Ther. 2011]
Review Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, Takeda S, Wilton SD, Wolff JA, Wooddell CI, Xiao X, et al. Mol Ther. 2011 May; 19(5):830-40. Epub 2011 Apr 5.
Review Therapeutic approaches to muscular dystrophy. [Hum Mol Genet. 2011]
Review Therapeutic approaches to muscular dystrophy.
Goyenvalle A, Seto JT, Davies KE, Chamberlain J. Hum Mol Genet. 2011 Apr 15; 20(R1):R69-78. Epub 2011 Mar 24.

See all...

Related information

Related Citations
Calculated set of PubMed citations closely related to the selected article(s) retrieved using a word weight algorithm. Related articles are displayed in ranked order from most to least relevant, with the “linked from” citation displayed first.
Compound (MeSH Keyword)
PubChem chemical compound records that are classified under the same Medical Subject Headings (MeSH) controlled vocabulary as the current articles.
Substance (MeSH Keyword)
PubChem chemical substance (submitted) records that are classified under the same Medical Subject Headings (MeSH) controlled vocabulary as the current articles.
Cited in PMC
Full-text articles in the PubMed Central Database that cite the current articles.

Recent activity

Clear Turn Off Turn On

Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how cl...
Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?
Trends Mol Med. 2006 Mar ;12(3):122-9. Epub 2006 Jan 27 .

PubMed
Do drug users in China who frequently receive detoxification treatment change th...
Do drug users in China who frequently receive detoxification treatment change their risky drug use practices and sexual behavior?
Drug Alcohol Depend. 2006 Sep 1 ;84(1):114-21. Epub 2006 Jan 26 .

PubMed
Tetracyclines: nonantibiotic properties and their clinical implications.
Tetracyclines: nonantibiotic properties and their clinical implications.
J Am Acad Dermatol. 2006 Feb ;54(2):258-65.

PubMed
Antivirals for influenza in healthy adults: systematic review.
Antivirals for influenza in healthy adults: systematic review.
Lancet. 2006 Jan 28 ;367(9507):303-13.

PubMed
Relationships between performance-based tests and patients' ratings of activity ...
Relationships between performance-based tests and patients' ratings of activity limitations, self-efficacy, and pain in fibromyalgia.
Arch Phys Med Rehabil. 2006 Feb ;87(2):259-64.

PubMed

Your browsing activity is empty.

Activity recording is turned off.

Turn recording back on

PubMed

Result Filters

Display Settings:

Send to:

Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?

Source

Abstract

Publication Types, MeSH Terms, Substances

Publication Types

MeSH Terms

Substances

LinkOut - more resources

Full Text Sources

Other Literature Sources

Medical

Molecular Biology Databases

Supplemental Content

Save items

Related citations in PubMed

Cited by 16 PubMed Central articles

Related information

Recent activity

Simple NCBI Directory

Getting Started

Resources

Popular

Featured

NCBI Information