Featured Research

from universities, journals, and other organizations

Gene responsible for Duchenne muscular dystrophy can be repaired, research shows

Date:
April 15, 2010
Source:
Université Laval
Summary:
Researchers have found that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy.

Researchers from Université Laval's Faculty of Medicine and the CHUQ Research Center have shown that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy.

Related Articles


The team, led by Professor Jacques P. Tremblay, is presenting its new therapeutic approach in an article published in the online version of the scientific journal Gene Therapy.

Duchenne muscular dystrophy is a hereditary disease affecting one in 3,500 males. It causes progressive muscle degeneration that begins in early childhood and causes death by age 25 in most people afflicted. The disease is caused by mutations that affect a protein called "dystrophin." The mutations alter the normal nucleotide sequences of this protein's gene and stop its synthesis.

Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes -- called meganucleases -- with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells. They repeated the experiment in vivo with mice carrying the mutation that causes the illness. Both series of testing showed that the meganucleases can lead to a restoration of the normal nucleotide sequences of the dystrophin gene and its expression in muscle cells.

A number of hurdles must be overcome before this approach can be tested in humans, cautions Dr. Tremblay. "It must first be proven in laboratory animals that it is possible to insert a meganuclease targeting the dystrophin gene directly into muscle cells, and that this will induce the synthesis of dystrophin able to attach to the muscle fiber membrane," explains the researcher. "We're still two to three years away from this stage," he estimates. "Subsequent stages, including human trials, could take even longer," adds Dr. Tremblay.

In addition to Jacques P. Tremblay, the study was co-authored by: Pierre Chapdelaine, Christophe Pichavant, and Joël Rousseau from the CHUQ Research Center; Frédéric Pâques from Cellectis.

Professor Tremblay's work is funded by the Canadian Institutes of Health Research, Fonds de recherche en santé du Québec, Ministère du Développement économique du Québec, Muscular Dystrophy Canada, the Amyotrophic Lateral Sclerosis Society of Canada, Association française contre les myopathies, Jesse's Journey, and the Neuromuscular Disease Foundation.


Story Source:

The above story is based on materials provided by Université Laval. Note: Materials may be edited for content and length.


Journal Reference:

  1. P Chapdelaine, C Pichavant, J Rousseau, F Pâques and J P Tremblay. Meganucleases can restore the reading frame of a mutated dystrophin. Gene Therapy, 2010; DOI: 10.1038/gt.2010.26

Cite This Page:

Université Laval. "Gene responsible for Duchenne muscular dystrophy can be repaired, research shows." ScienceDaily. ScienceDaily, 15 April 2010. <www.sciencedaily.com/releases/2010/04/100415110005.htm>.
Université Laval. (2010, April 15). Gene responsible for Duchenne muscular dystrophy can be repaired, research shows. ScienceDaily. Retrieved November 26, 2014 from www.sciencedaily.com/releases/2010/04/100415110005.htm
Université Laval. "Gene responsible for Duchenne muscular dystrophy can be repaired, research shows." ScienceDaily. www.sciencedaily.com/releases/2010/04/100415110005.htm (accessed November 26, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Wednesday, November 26, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Pet Dogs to Be Used in Anti-Ageing Trial

Pet Dogs to Be Used in Anti-Ageing Trial

Reuters - Innovations Video Online (Nov. 26, 2014) — Researchers in the United States are preparing to discover whether a drug commonly used in human organ transplants can extend the lifespan and health quality of pet dogs. Video provided by Reuters
Powered by NewsLook.com
Today's Prostheses Are More Capable Than Ever

Today's Prostheses Are More Capable Than Ever

Newsy (Nov. 26, 2014) — Advances in prosthetics are making replacement body parts stronger and more lifelike than they’ve ever been. Video provided by Newsy
Powered by NewsLook.com
From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

Newsy (Nov. 25, 2014) — The US FDA is announcing new calorie rules on Tuesday that will require everywhere from theaters to vending machines to include calorie counts. Video provided by Newsy
Powered by NewsLook.com
Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Newsy (Nov. 25, 2014) — Need another reason to eat yogurt every day? Researchers now say it could reduce a person's risk of developing type 2 diabetes. 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:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

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