Iconocast Logo

Welcome To Iconocast

How to add a URL link from your web site to the Iconocast web sites

Virtual tour of Southern California
blank

 

Recent News and Articles on the Keywords: muscular dystrophy + new target + new  Related to the article below (Last Update: 5/5/2008)

Researchers Discover Molecular Basis Of A Form Of Muscular Dystrophy
Science Daily (press release) - Apr 30, 2008
... for creating this protein, scientists could develop new drugs to stop muscle wasting from limb-girdle muscular dystrophy and other conditions. ...

AFP
Muscular dystrophy type linked to missing protein: study
AFP - Apr 30, 2008
... giving them more insight into the causes of limb-girdle muscular dystrophy. They hope the findings of their research will lead to the development of new ...
Summit Reports Progress of Duchenne Muscular Dystrophy Programme ...
PharmaLive.com (press release), PA - Apr 29, 2008
These latest data were presented to leading research scientists and companies in the field of neuromuscular diseases at the New Directions in Muscle Biology ...LON:SUMM

Economist
Gene therapy Seeing is believing
Economist, UK - May 1, 2008
For diseases such as cystic fibrosis or muscular dystrophy, which involve one or a few inherited genetic changes, clinical trials are attempting to ...
PTC Therapeutics Announces Initiation of Phase 2b Registration ...
FOXBusiness - Apr 23, 2008
Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle disorders that cause the loss of both muscle function and independence. ...
A passion for controversy
New Jersey Herald, NJ - Apr 28, 2008
An avid dancer, in 1982 he boogied a 24-hour marathon at the Rockaway Mall to raise money for muscular dystrophy research. At FDU, he was a member of the ...
AVI BioPharma Announces Dr. Ryszard Kole as Senior Vice President ...
CNNMoney.com - Apr 16, 2008
AVI's ESPRIT technology is initially being applied to potential treatments for Duchenne muscular dystrophy. AVI's NeuGene compounds are also designed to ...AVII
Neighbors applaud your achievements
Appleton Post Crescent,  USA - Apr 27, 2008
The award money will be used to support a new strategy that addresses the lack of donated vehicles. "We will now be able to target families who have been on ...
Sports fans who have it tougher
The Argus.co.uk, UK - Apr 11, 2008
She was so impressed she moved her family from Hertfordshire so that Matthew, who has duchenne muscular dystrophy, could go there. Pupil Tim Hayes, 14, ...
Insmed Provides Update on Follow-On Biologics and IPLEX(TM) Programs
PR Newswire (press release), NY - Apr 29, 2008
The drug is also being investigated for various other indications with unmet medical needs including Myotonic Muscular Dystrophy (MMD) and Amyotrophic ...INSM
Source: Google News

A new retrotransposable human L 1 element from the LRE 2 locus on chromosome 1 q produces a … -
SE Holmes, BA Dombroski, CM Krebs, CD Boehm, HH … - Nature Genetics, 1994 - nature.com
... by a nick at the chromosomal target site: a ... basis for Duchenne versus Becker muscular
dystrophy: correlation of ... Cold Spring Harbor Laboratory, New York, 1982). ...

Muscle biopsy -
V Dubowitz - A Practical Approach - intl.elsevierhealth.com
... be of immense value beyond its target readership." BMA ... What's New: Presents all the
latest techniques in ... disorders I: Duchenne and Becker muscular dystrophy 11. ...

Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification -
JS Chamberlain, RA Gibbs, JE Ranier, PN Nguyen, CT … - Nucleic Acids Res, 1988 - Oxford Univ Press
... all cases result from a new mutation (2 ... in the Treatment of Duchenne's Muscular
Dystrophy N. Engl ... CR Cantor Multiplex allele-specific target amplification based ...

Glycosylation defects: a new mechanism for muscular dystrophy? -
PK Grewal, JE Hewitt - Human Molecular Genetics, 2003 - Oxford Univ Press
... Different target specificities could account for differences in ... 3-q13.1 is a new
distinct member ... glycosyltransferase, in an animal model of muscular dystrophy. ...

Exclusive paternal origin of new mutations in Apert syndrome -
DM Moloney, SR Slaney, M Oldridge, SA Wall, P … - Nature Genetics, 1996 - nature.com
... containing the hprt cDNA as target for mutagenesis. ... and point mutations in Duchenne
muscular dystrophy: most deletions ... Paternal origin of new mutations in Von ...

MLPA and MAPH: New techniques for detection of gene deletions -
LN Sellner, GR Taylor - Human Mutation, 2004 - doi.wiley.com
... of detected mutations, such as Duchenne muscular dystrophy, spinal muscular atrophy,
Charcot ... RECENT DETECTION METHODS Recently, two new methods have been ...

… an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy -
EA Burton, JM Tinsley, PJ Holzfeind, NR Rodrigues, … - Proceedings of the National Academy of Sciences, 1999 - National Acad Sciences
Medical Sciences A second promoter provides an alternative target for therapeutic
up-regulation of utrophin in Duchenne muscular dystrophy. ...

Defective glycosylation in muscular dystrophy -
F Muntoni, M Brockington, DJ Blake, S Torelli, SC … - The Lancet, 2002 - Elsevier
... This opens up new avenues of research. ... other as yet uncharacterised forms of muscular
dystrophy and neuronal ... skeletal muscle is not a primary target in CDG ...

Muscular Dystrophy and Neuronal Migration Disorder Caused by Mutations in a Glycosyltransferase, … -
A Yoshida, K Kobayashi, H Manya, K Taniguchi, H … - Developmental Cell, 2001 - Elsevier
... Our findings suggest a new pathomechanism, in which ... a-dystroglycan is a potential
target relevant for ... manifestations as well as muscular dystrophy in general. ...

A New Clinical Condition Linked to a Novel Mutation in Lamins A and C with Generalized Lipoatrophy, … -
F Caux, E Dubosclard, O Lascols, B Buendia, O … - Journal of Clinical Endocrinology & Metabolism, 2003 - Endocrine Soc
... tissue seems to be the main target of the ... G Bonne, and X Ferrer A new mutation of ...
to autosomal dominant axonal neuropathy, muscular dystrophy, cardiac disease ...

Source: Google Scholar

New Target For Muscular Dystrophy Drug Therapy
Researchers at the University of Pennsylvania School of Medicine report how the gene for utrophin, which codes for a protein very similar to dystrophin, the defective protein in Duchenne muscular dystrophy (DMD), puts the brakes on its own expression in muscle cells, thereby suggesting a new target for treatment. The findings were published online in Molecular Biology of the Cell, in advance of print publication.

The production of utrophin slows in fetal muscles soon after birth, after which dystrophin takes over as the primary muscle-associated protein. How this normal utrophin silencing occurs has been a mystery, until now. If the brakes on utrophin production could be removed by drug intervention, then increased utrophin expression could substitute for dystrophin as a possible therapy for DMD, which affects 1 in 3,500 males.
Utrophin is normally made at the junction where nerves meet muscles, an area called the neuromuscular junction or synapse. In the present study, the Penn team discovered that silencing is applied by a protein called Ets-2 repressor factor (ERF) sitting on a small piece of the utrophin gene called the N-box.

"We demonstrated that ERF significantly reduces or represses the activity of utrophin's N-box in muscle cells of mice," says senior author Tejvir S. Khurana, MD, PhD, Associate Professor of Physiology and Member of the Pennsylvania Muscle Institute. When the N-box was deleted from the utrophin gene, ERF had no effect on silencing the utrophin gene, as measured by an increase in utrophin gene-promoter activity. In another experiment in which ERF was repressed, the researchers found utrophin mRNA production increased.

"This approach of 'repressing the repressor' is medically relevant to treating muscular dystrophy in that we hope to one day be able to upregulate utrophin production," explains Khurana.

Because utrophin is over 80 percent identical to dystrophin in its gene sequence, utrophin could substitute for it in muscle cells. In normal muscle cells dystrophin is part of a large complex of proteins that attaches muscle cells to surrounding tissues. In DMD muscle cells, dystrophin cannot perform this function and the muscles slowly fall apart. DMD patients begin to have muscle weakness and motor difficulties as children, and the condition worsens with age, eventually proving fatal around the third decade of life.

"Dr. Khurana's work hints at what could be an important new drug target for DMD�"the more options we have with this disease, the better," says Sharon Hesterlee, PhD, Vice President for Translational Research at the Muscular Dystrophy Association. "We've known for a while that increasing utrophin expression can reduce symptoms of the disease, but it's very difficult to use a drug to increase gene activity. What's nice about this work is that now we can try to 'block a blocker' to get the same effect�"it's a more drug-friendly approach."
Other therapeutic strategies for DMD involve muscle-cell implantation, stem-cell treatment, and gene therapy. While there has been some progress with these approaches, there have been many difficulties with graft vs. host rejection and gene delivery. This new research suggests that blocking ERF, either with drugs or by interfering with its RNA, may be more generally feasible in most DMD patients.

There are several animal models of DMD, most notably the mdx mouse. Khurana and his colleagues are currently investigating whether repressing ERF in mdx mouse muscle reduces muscle deterioration.

"We have worked on this problem for a number of years, and our current findings are a logical incremental step in understanding how utrophin could become an effective tool for treating DMD," states Khurana. He cautions that while he hopes his work will lead to an effective treatment someday, there are many steps and hurdles to get through first.

This work was funded in part by grants from the National Institute of Arthritis, Musculoskeletal, and Skin Disease, the National Eye Institute, the Muscular Dystrophy Association, and the Canadian Institute of Health Research. Co-authors on the study are Kelly J. Perkins, Utpal Basu, Murat T. Budak, Caroline Ketterer, Santhosh M. Baby, Olga Lozynska and Neal A. Rubinstein, from Penn, along with John A. Lunde and Bernard J. Jasmin of the University of Ottawa.

PENN Medicine is a $2.9 billion enterprise dedicated to the related missions of medical education, biomedical research, and high-quality patient care. PENN Medicine consists of the University of Pennsylvania School of Medicine (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System.

Penn's School of Medicine is ranked #2 in the nation for receipt of NIH research funds; and ranked #3 in the nation in U.S. News & World Report's most recent ranking of top research-oriented medical schools. Supporting 1,400 fulltime faculty and 700 students, the School of Medicine is recognized worldwide for its superior education and training of the next generation of physician-scientists and leaders of academic medicine.

The University of Pennsylvania Health System includes three hospitals, all of which have received numerous national patient-care honors [Hospital of the University of Pennsylvania; Pennsylvania Hospital, the nation's first hospital; and Penn Presbyterian Medical Center]; a faculty practice; a primary-care provider network; two multispecialty satellite facilities; and home care and hospice.

University of Pennsylvania School of Medicine
3600 Market St., Ste 240
Philadelphia, PA 19104
United States
http://www.med.upenn.edu
 
 
Google
Web www.iconocast.com

Search inside Iconocast for the keyword you have in mind.

Iconocast has collected more than 50,000 articles and press releases on health and science.

These are current and most up to date press releases on the subject you are searching.

We collect current health and science press releases daily from more than 5000 research and health institutes. Here is an example : The elderberry way to perfect skin

We believe if you do search inside Iconocast, you will get better results than searching the web alone.

 
 
Continue News With: News9A


ADVERTISEMENT

Iconocast is about learning and teaching without borders; we offer eMarketing, Internet Advertising, Internet Marketing, Search Engine Optimization, Search Engine Marketing, Online Branding, and eMarketing News Services.

 

Iconocast Home Page

Contact Iconocast

© 2003-07. ICONOCAST is a trademark of iconocast.com.