About My Health Careers Internship MedBlogs Contact us
Medindia LOGIN REGISTER
Advertisement

Age Matters in Regenerating Muscle in Duchenne Muscular Dystrophy

by Bidita Debnath on April 15, 2014 at 11:13 PM
Font : A-A+

 Age Matters in Regenerating Muscle in Duchenne Muscular Dystrophy

A team of scientists have published details of how a class of drugs called "HDACis" drive muscle-cell regeneration in the early stages of dystrophic muscles, but fail to work in late stages.

The study was led by Pier Lorenzo Puri, M.D., associate professor at Sanford-Burnham Medical Research Institute (Sanford-Burnham), in collaboration with Fondazione Santa Lucia in Rome, Italy. The findings are key to furthering clinical development of HDACis for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting disease.

Advertisement

A symphony to rebuild muscle

The research, to be published April 15 in Genes and Development, used mouse models of DMD to show how fibro-adipogenic progenitor cells (FAPs) act like orchestra conductors in the music of muscle regeneration. FAPs sit in the space between muscle fibers and coordinate a complex symphony—receiving the notes that muscle has been damaged and directing muscle stem cells—satellite cells—to rebuild muscle.
Advertisement

"HDACis create an environment conducive for FAPs to direct muscle regeneration—but only during the early stages of DMD progression in mice," said Puri. "At some point, DMD progresses to a pathological point of no return and become permanently resistant to muscle-regeneration cures and to HDACis.

HDACis open the blueprints for muscle regeneration

HDACis stands for histone deacetylase inhibitors. They are epigenetic drugs that work by facilitating the accessibility to the genes that code for muscle proteins by the cell machinery that transcribes the genetic code into proteins. In essence, HDACis open the blueprints for protein manufacturing and instruct FAPs to support muscle regeneration.

In normal wear and tear, FAPs direct stem cells within the muscle to regenerate and repair damaged muscle. In DMD, the persistent breakdown of muscle cells creates an environment in such disarray that FAP's ability to direct muscle regeneration is compromised—like trying to conduct a symphony with punk rock music in the background.

Collaborating to find a treatment for DMD

Puri, along with his Italian colleagues at Fondazione Santa Lucia, Italfarmaco, and Parent Project Muscular Dystrophy, an advocacy association, are currently developing HDACis for the treatment of DMD - a clinical trial with DMD boys is currently ongoing.

"Our study is important because it provides the rational for the clinical development of HDACis to treat DMD," said Puri. "And, now that we understand the mechanics and sensitivities of the muscle-regeneration system, we have the rationale and can use new tools to select patients most likely to benefit from HDACIs based on their FAP profile, predict outcomes, and see how long patients should remain on the therapy."

"Duchenne muscular dystrophy patients and their families rely on important research such as that performed by Dr. Puri," said Debra Miller, Founder of Cure Duchenne, a patient advocacy group. "Our efforts at Cure Duchenne are to support leading scientists in the world to bring life-saving drugs to help this generation of Duchenne boys, and our vision is to cure Duchenne muscular dystrophy. Every added piece of knowledge about the disease brings us closer to realizing our goals."

Source: Eurekalert
Advertisement

Advertisement
News A-Z
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
News Category
What's New on Medindia
Health Benefits of Sea Buckthorn
Contraceptive Pills in Polycystic Ovary Syndrome (PCOS) Curtail Type 2 Diabetes Risk
Mushroom May Help Cut Down the Odds of Developing Depression
View all

Medindia Newsletters Subscribe to our Free Newsletters!
Terms & Conditions and Privacy Policy.

More News on:
Muscular Dystrophy 

Recommended Reading
Light may Help Artificially Control Muscles
Scientists have discovered a new method to artificially control muscles using light, with the ......
Stronger Muscles in Teens Linked With Lower Risk of Diabetes, Heart Disease
Researchers who studied the effect of muscle strength in sixth grade boys and girls found that ......
That Slim and Fit Neighbour also Has Better Muscles Than You
Some people stay slim and trim without much effort and despite eating the same high-calorie food ......
Powerful Artificial Muscles Created With the Help of Fishing Lines
An international group of experts revealed that they have managed to create inexpensive artificial ....
Muscular Dystrophy
Overview about muscular dystrophy, a muscle disease characterized by muscle weakness and muscle wast...

Disclaimer - All information and content on this site are for information and educational purposes only. The information should not be used for either diagnosis or treatment or both for any health related problem or disease. Always seek the advice of a qualified physician for medical diagnosis and treatment. Full Disclaimer

© All Rights Reserved 1997 - 2021

This site uses cookies to deliver our services. By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use