Navigation Links
Exon skipping to restore gene expression is promising therapeutic strategy for muscular dystrophy
Date:1/15/2013

New Rochelle, NY, January 15, 2013A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protein production. Two innovative examples of this strategy used to correct gene defects associated with muscular dystrophy are described in articles in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website.

Willem Hoogaars and a team of researchers from France, the Netherlands, Finland, and Germany evaluated a combination of an exon-skipping treatment delivered via an adeno-associated virus (AAV) vector and a compound that inhibits myostatin signaling in a mouse model of Duchenne muscular dystrophy. The dual treatments led to improved muscle force and a decrease in ineffective muscle contractions, as well as increased body weight, muscle mass, and muscle fiber size. They did not, however, improve the effects of each treatment given individually. The results are reported in the article "Combined Effect of AAV-U7-Induced Dystrophin Exon Skipping and Soluble Activin Type IIB Receptor in mdx Mice."

Francesca Gualandi and colleagues, University of Ferrara and University of Padua, Italy, used exon skipping to inactivate mutated RNA messages responsible for defective collagen structure and function in muscle tissue affected by Ullrich muscular dystrophy or Bethlem myopathy. The authors describe their approach in "Antisense-Induced Messenger Depletion Corrects a COL6A2 Dominant Mutation in Ullrich Myopathy."

"This combines two very promising therapeutic approaches in a relevant animal model of muscular dystrophy," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.


'/>"/>

Contact: Vicki Cohn
vcohn@liebertpub.com
914-740-2100 x2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

Related biology technology :

1. Brain-activated muscle stimulation restores monkeys hand movement after paralysis
2. Veracyte Announces Medicare Coverage for Its Afirma® Gene Expression Classifier for Improved Thyroid Nodule Diagnosis
3. HemoShear Partners with Expression Analysis (EA) to Develop a Human Drug-Induced Vascular Response Database
4. Genedata Expressionist® Established as Data Analysis Platform for Oncological Research
5. S*BIOs Novel JAK2 Inhibitor Pacritinib (SB1518) and Histone Deacetylase Inhibitor Pracinostat (SB939) Demonstrate Promising Activity in Phase 2 Studies for Patients with Myelofibrosis (MF)
6. New 3-D transistors promising future chips, lighter laptops
7. Founder of National Childrens Leukemia Unveiled Non-Embryonic Stem Cell Treatment Promising Cure for Cancer at GIL 2012: Europe
8. University of Miami study finds RBAC to possess promising immunomodulating effects with respect to natural killer cell and cytokine activity
9. Promising Data Presented on GenVec Malaria Program
10. Bioprinting has promising future
11. Cell Therapeutics, Inc. Reports Results of Annual Meeting of Shareholders
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Exon skipping to restore gene expression is promising therapeutic strategy for muscular dystrophy
(Date:6/23/2016)... , ... June 23, 2016 , ... ... announced the launch of the Supplyframe Design Lab . Located in Pasadena, ... explore the future of how hardware projects are designed, built and brought to ...
(Date:6/23/2016)... Calif. , June 23, 2016  Blueprint Bio, ... biological discoveries to the medical community, has closed its ... Matthew Nunez . "We have received ... with the capital we need to meet our current ... essentially provide us the runway to complete validation on ...
(Date:6/23/2016)... ... June 23, 2016 , ... Regulatory ... technical consulting, provides a free webinar on Performing Quality Investigations: Getting ... at 12pm CT at no charge. , Incomplete investigations are still a major ...
(Date:6/22/2016)... 2016 Cell Applications, Inc. and StemoniX ... produce up to one billion human induced pluripotent ... week. These high-quality, consistent stem cells enable researchers ... spend more time doing meaningful, relevant research. This ... manufacturing process that produces affordable, reliable HiPSC for ...
Breaking Biology Technology:
(Date:6/22/2016)... , June 22, 2016  The American College of Medical ... Show Executive Magazine as one of the fastest-growing trade ... 25-27 at the Bellagio in Las Vegas ... highest percentage of growth in each of the following categories: ... companies and number of attendees. The 2015 ACMG Annual Meeting ...
(Date:6/16/2016)... The global Biometric ... USD 1.83 billion by 2024, according to a ... proliferation and increasing demand in commercial buildings, consumer ... the market growth.      (Logo: ... of advanced multimodal techniques for biometric authentication and ...
(Date:6/3/2016)... June 3, 2016 ... Nepal hat ein ... hochsicherer geprägter Kennzeichen, einschließlich Personalisierung, Registrierung und ... der Produktion und Implementierung von Identitätsmanagementlösungen. Zahlreiche ... im Januar teilgenommen, aber Decatur wurde als ...
Breaking Biology News(10 mins):