Navigation Links
Researchers prove the gene responsible for Duchenne muscular dystrophy can be repaired

This release is available in French.

Quebec City, April 15, 2010Researchers from Universit Laval's Faculty of Medicine and the CHUQ Research Center have proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy. The team, led by Professor Jacques P. Tremblay, is presenting its new therapeutic approach in an article published today 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 enzymescalled meganucleaseswith 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.


Contact: Jean-Franois Hupp
Universit Laval

Related biology news :

1. Pitt Dental School researchers find susceptibility for caries, gum disease in genes
2. Hopkins researchers put proteins right where they want them
3. EVMS researchers identify potential target for treatment of obesity-related diseases
4. UI researchers analyze implications of intelligent design for human behavior
5. Stanford researchers find electrical current stemming from plants
6. UCLA researchers make first direct recording of mirror neurons in human brain
7. MIT researchers harness viruses to split water
8. Researchers use novel nanoparticle vaccine to cure type 1 diabetes in mice
9. Heinz Maier-Leibnitz Prizes 2010: 6 outstanding young researchers honored
10. Smart orthopedic implants and self-fitting tissue scaffolding created by UMMS researchers
11. Researchers find a better way to track stem cells
Post Your Comments:
(Date:11/12/2015)... 2015  A golden retriever that stayed healthy despite ... has provided a new lead for treating this muscle-wasting ... Institute of MIT and Harvard and the University of ... Cell, pinpoints a protective gene that ... effects. The Boston Children,s lab of Lou Kunkel ...
(Date:11/11/2015)... MedNet Solutions , an innovative SaaS-based eClinical technology company that ... announce that it will be a Sponsor of the ... held November 17-19 in Hamburg , Germany.  ... iMedNet , MedNet,s easy-to-use, proven and affordable eClinical ... able to deliver time and cost savings of up to ...
(Date:11/9/2015)... 09, 2015 ... the "Global Law Enforcement Biometrics Market ... --> ) has announced the ... Biometrics Market 2015-2019" report to their ... ( ) has announced the addition ...
Breaking Biology News(10 mins):
(Date:11/24/2015)... FAR HILLS, N.J. (PRWEB) , ... November 24, 2015 , ... ... University, as the recipient of the 2016 USGA Green Section Award. Presented annually since ... of golf through his or her work with turfgrass. , Clarke, of ...
(Date:11/24/2015)... ... 2015 , ... International Society for Pharmaceutical Engineering (ISPE) closed ... events for pharmaceutical manufacturing: 2015 Annual Meeting. The conference took place in Philadelphia, ... number of attendees in more than a decade. , “The 2015 Annual ...
(Date:11/24/2015)... ... November 24, 2015 , ... The Academy of Model ... Group (SIG), MultiGP, also known as Multirotor Grand Prix, to represent the First–Person View ... years. Many AMA members have embraced this type of racing and several new model ...
(Date:11/24/2015)... FRANCISCO , Nov. 24, 2015  Twist ... announced that Emily Leproust, Ph.D., Twist Bioscience chief ... Jaffray Healthcare Conference on December 1, 2015 at ... Hotel in New York City. --> ... . Twist Bioscience is on Twitter. ...
Breaking Biology Technology: