Navigation Links
Researchers prove the gene responsible for Duchenne muscular dystrophy can be repaired
Date:4/15/2010

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
jean-francois.huppe@dc.ulaval.ca
418-656-7785
Universit Laval
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:6/14/2017)...  IBM (NYSE: IBM ) is introducing several innovative ... to developing collaboration between startups and global businesses, taking place ... the event, nine startups will showcase the solutions they have ... France is one ... a 30 percent increase in the number of startups created ...
(Date:5/16/2017)... TEANECK, N.J. , May 16, 2017  Veratad ... leading provider of online age and identity verification solutions, ... the K(NO)W Identity Conference 2017, May 15 thru May ... Ronald Regan Building and International Trade Center. ... across the globe and in today,s quickly evolving digital ...
(Date:5/6/2017)... SINGAPORE , May 5, 2017 ... has just announced a new breakthrough in biometric ... that exploits quantum mechanical properties to perform ... new smart semiconductor material created by Ram Group ... across finance, entertainment, transportation, supply chains and security. ...
Breaking Biology News(10 mins):
(Date:10/11/2017)... INDIANAPOLIS , Oct. 11, 2017  VMS BioMarketing, a ... of a nationwide oncology Clinical Nurse Educator (CNE) network, which ... growing need for communication among health care professionals to enhance ... physicians, nurses, office staff, and other health care professionals to ... for breast cancer. ...
(Date:10/11/2017)... ... October 11, 2017 , ... ... and pregnancy rates in frozen and fresh in vitro fertilization (IVF) ... maternal age to IVF success. , After comparing the results from the fresh ...
(Date:10/10/2017)... Angeles, CA (PRWEB) , ... ... ... Inc., a development-stage cancer-focused pharmaceutical company advancing targeted antibody-drug conjugate (ADC) therapeutics, ... uses of targeted HPLN (Hybrid Polymerized Liposomal Nanoparticle), a technology developed in ...
(Date:10/10/2017)... firm Parks Associates announced today that Tom Kerber , ... Meeting , October 11 in Scottsdale, Arizona . Kerber ... smart safety and security products impact the competitive landscape. ... Parks Associates: Smart Home Devices: Main Purchase Driver ... "The residential security market has experienced continued growth, and the introduction ...
Breaking Biology Technology: