Featured Research

from universities, journals, and other organizations

Researchers identify novel therapy to treat muscular dystrophy

Date:
June 25, 2013
Source:
Boston University College of Arts & Sciences
Summary:
Researchers have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model.

Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model. The findings, published in Human Molecular Genetics, represent a paradigm shift for the treatment of muscular dystrophy as well as a host of other disabling and devastating muscle diseases.

The study was led by Mahasweta Girgenrath, PhD, assistant professor and director of the Muscle Disorders and Regenerative Biology Laboratory at BU Sargent College's Department of Health Sciences. Boston University (BU) researchers and postdoctoral fellows Jenny Yamauchi, Ajay Kumar, Lina Duarte, and Thomas Mehuron were collaborators on this study.

Muscular Dystrophy type 1A (MDC1A) is the second most common form of congenital muscular dystrophy. Patients with this disease have poor muscle tone at birth, extremely compromised neuromuscular function, and are rarely able to walk independently. Most patients with MDC1A succumb to a premature death due to either respiratory complications or failure to thrive. Although significant strides have been made towards understanding the molecular and biochemical mechanisms underlying MDC1A, there remains no effective therapy in place to combat this lethal disease.

The research team, led by Girgenrath, hypothesized that the complex pathology seen in MDC1A may be the result of dysregulation of multiple cellular functions and processes, meaning that strategies which simultaneously target several of those mechanisms might lead to a reduction of symptoms.

"Very few studies have utilized the power of combinatorial therapy in the context of muscular dystrophy." said Professor Girgenrath, the study's corresponding author. "While most MD treatments are single-target therapies, we're delving into combinations of different therapies to target multiple pathways."

The research team studied the outcome of combining the following single mode treatments: increasing regeneration, by overexpressing muscle specific insulin like growth factor-1, IGF-1 and preventing cell death, by inhibiting the expression of Bax, a pro-apoptotic protein. In addition, to test the translational potential of this combination therapy, the researchers systemically treated Bax deficient dystrophic mice with recombinant human IGF-1 (IPLEX TM, manufactured by Insmed Inc).

By combining these two therapies, researchers found that in addition to increased body and muscle weight, mice showed enhanced locomotory capacities and remarkable improvement in muscle pathology. The most impressive outcome was the significant resolution of inflammation and fibrosis, not seen with single mode therapies. The research team concluded that the use of this combination therapy is an effective treatment for MDC1A, highlighting a compelling argument that a combinatorial approach has a synergistic benefit and could have the potential of treating patients with congenital muscular dystrophy.

Research highlighted in this news release was funded in part by Cure CMD, Struggle against Muscular Dystrophy (S.A.M), and the Muscular Dystrophy Association (MDA).


Story Source:

The above story is based on materials provided by Boston University College of Arts & Sciences. Note: Materials may be edited for content and length.


Journal Reference:

  1. J. Yamauchi, A. Kumar, L. Duarte, T. Mehuron, M. Girgenrath. Triggering Regeneration and Tackling Apoptosis: A Combinatorial Approach to Treating MDC1A. Human Molecular Genetics, 2013; DOI: 10.1093/hmg/ddt280

Cite This Page:

Boston University College of Arts & Sciences. "Researchers identify novel therapy to treat muscular dystrophy." ScienceDaily. ScienceDaily, 25 June 2013. <www.sciencedaily.com/releases/2013/06/130625161858.htm>.
Boston University College of Arts & Sciences. (2013, June 25). Researchers identify novel therapy to treat muscular dystrophy. ScienceDaily. Retrieved April 17, 2014 from www.sciencedaily.com/releases/2013/06/130625161858.htm
Boston University College of Arts & Sciences. "Researchers identify novel therapy to treat muscular dystrophy." ScienceDaily. www.sciencedaily.com/releases/2013/06/130625161858.htm (accessed April 17, 2014).

Share This



More Health & Medicine News

Thursday, April 17, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Could Even Casual Marijuana Use Alter Your Brain?

Could Even Casual Marijuana Use Alter Your Brain?

Newsy (Apr. 16, 2014) A new study conducted by researchers at Northwestern and Harvard suggests even casual marijuana use can alter your brain. Video provided by Newsy
Powered by NewsLook.com
Thousands Of Vials Of SARS Virus Go Missing

Thousands Of Vials Of SARS Virus Go Missing

Newsy (Apr. 16, 2014) A research institute in Paris somehow misplaced more than 2,000 vials of the deadly SARS virus. Video provided by Newsy
Powered by NewsLook.com
Formerly Conjoined Twins Released From Dallas Hospital

Formerly Conjoined Twins Released From Dallas Hospital

Newsy (Apr. 16, 2014) Conjoined twins Emmett and Owen Ezell were separated by doctors in August. Now, nearly nine months later, they're being released from the hospital. Video provided by Newsy
Powered by NewsLook.com
Ebola Outbreak Now Linked To 121 Deaths

Ebola Outbreak Now Linked To 121 Deaths

Newsy (Apr. 15, 2014) The ebola virus outbreak in West Africa is now linked to 121 deaths. Health officials fear the virus will continue to spread in urban areas. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins