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:10/11/2017)... NJ (PRWEB) , ... October 11, 2017 , ... Personal eye wash is a basic ... one eye at a time. So which eye do you rinse first if a dangerous ... have Plum Duo Eye Wash with its unique dual eye piece. , “Whether ...
(Date:10/11/2017)... LAGUNA HILLS, Calif. , Oct. 11, 2017  SkylineDx ... London (ICR) and University of Leeds ... to risk-stratify patients with multiple myeloma (MM), in a multi-centric ... The University of Leeds is the ... UK, and ICR will perform the testing services to include ...
(Date:10/10/2017)... ... October 10, 2017 , ... San Diego-based team building and ... rebranding initiative announced today. The bold new look is part of a transformation ... moves into a significant growth period. , It will also expand its service offering ...
(Date:10/10/2017)... International research firm Parks Associates announced today that ... TMA 2017 Annual Meeting , October 11 in Scottsdale, ... security market and how smart safety and security products impact the competitive ... Parks Associates: Smart Home Devices: Main ... "The residential security market has experienced continued ...
Breaking Biology Technology:
(Date:4/11/2017)... PALM BEACH GARDENS, Fla. , April 11, ... biometric identity management and secure authentication solutions, today ... million contract by Intelligence Advanced Research Projects Activity ... technologies for IARPA,s Thor program. "Innovation ... the onset and IARPA,s Thor program will allow ...
(Date:4/11/2017)... No two people are believed to ... York University Tandon School of Engineering and Michigan ... partial similarities between prints are common enough that ... and other electronic devices can be more vulnerable ... in the fact that fingerprint-based authentication systems feature ...
(Date:4/6/2017)... LONDON , April 6, 2017 ... Control, RFID, ANPR, Document Readers, by End-Use (Transportation & ... Energy Facility, Oil, Gas & Fossil Generation Facility, Nuclear ... Healthcare, Educational, Other) Are you looking for ... Authentication sector? ...
Breaking Biology News(10 mins):