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 i
'/>"/>

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

Page: 1 2

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:1/22/2015)... 22, 2015 Pipette.com has added the ... their comprehensive portfolio of Eppendorf products. , The Eppendorf ... a Centrifuge 5424/5424 R and receive the following:, ... Research plus or Eppendorf Reference 2 , ...
(Date:1/22/2015)... 2015 Diagenode, Inc., a leading ... Bioruptor® and complete solutions for epigenetics research, recently ... alleviating the need for manual processing. The new ... ChIP of histones or transcription factors and a ...
(Date:1/22/2015)... 22, 2015  Derma Sciences, Inc. (Nasdaq: ... advanced wound care, announces that AMNIOEXCEL® and AMNIOMATRIX®, ... added to the Premier, Inc. Regenerative Skin Grafting ... the AMNIOEXCEL® and AMNIOMATRIX® product lines, which continue ...
(Date:12/24/2014)... Wash. , Dec. 24, 2014   BioLife Solutions , ... and marketer of proprietary clinical grade hypothermic storage ... shipping products for cells and tissues  ("BioLife" or the ... Meeting of Stockholders on May 4, 2015 (the "Annual Meeting"). ...
Breaking Biology Technology:Eppendorf Announces Promotional Bundle Which Includes: Eppendorf Centrifuge 5424, 3-Pack of Pipettes, and Tips - Available Now at Pipette.com 2Diagenode Launches Unique Semi-Automated Solution for Chromatin Immunoprecipitation 2Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 2Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 3Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 4Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 5BioLife Solutions Sets Date for Annual Meeting of Stockholders 2
... Inc. (OTC Bulletin Board Symbol: CCEL) (the "Company"), ... cord blood banks, today,announced results for fiscal 2007., ... November 30, 2007 were,approximately $17.5 million, up 2% ... November 30, 2006. The Company reported a net ...
... 11 AGA Medical Corporation ("AGA"),announced today it received ... ("ADO II"). AGA also announced the immediate availability,and launch ... the AGA family of,occlusion devices designed to occlude or ... heart defect that occurs when a blood vessel,called the ...
... Feb. 11 Genta,Incorporated (Nasdaq: GNTA ) ... with institutional investors to sell 6 million shares,of ... proceeds totaling,approximately $3.1 million before fees and expenses. ... about February 13, 2008, subject to the,satisfaction of ...
Cached Biology Technology:Cryo-Cell International, Inc. Reports Results for Fiscal 2007 2Cryo-Cell International, Inc. Reports Results for Fiscal 2007 3Cryo-Cell International, Inc. Reports Results for Fiscal 2007 4Cryo-Cell International, Inc. Reports Results for Fiscal 2007 5Cryo-Cell International, Inc. Reports Results for Fiscal 2007 6AGA Medical Corporation Receives European CE Mark Approval for the AMPLATZER(R) Duct Occluder II 2Genta Announces Common Stock Offering of Approximately $3.1 Million 2Genta Announces Common Stock Offering of Approximately $3.1 Million 3
(Date:1/22/2015)... , Jan. 21, 2015  Analyst Report Issued by Small ... Brands report showed that planet-wide, transactions at merchants on the ... the total number of credit, debit, and prepaid cards reached ... were victims of fraudulent card usage in 2012, and credit ...
(Date:12/24/2014)... 2014  Since its launch in December 2014, the 1U™ ... pain of trying to remember their usernames and passwords through ... their smartphones. To assist people who have struggled to remember ... created 1U and focuses on redefining identity, announced today that ...
(Date:12/19/2014)... 18, 2014 Research and Markets ( http://www.researchandmarkets.com/research/86ncd6/micro_market ) ... North America Perimeter Security Systems Market" report to their ... The North American perimeter security market is estimated to grow ... the U.S. market holds a larger share in this market, ...
Breaking Biology News(10 mins):$4.80 Near Term Price Target with "Buy" given by CapRock Research for NXT-ID, Inc. (NASDAQ: NXTD) issued by Small Cap IR 21U Offers Best Solution to the Username / Password Dilemma: For FREE! 21U Offers Best Solution to the Username / Password Dilemma: For FREE! 3Micro Market Monitor: North America Perimeter Security Systems Market (Intrusion Detection Sensor, Video Surveillance, Communication/Alarm and Notification, Access Control System) 2Micro Market Monitor: North America Perimeter Security Systems Market (Intrusion Detection Sensor, Video Surveillance, Communication/Alarm and Notification, Access Control System) 3
... to know if your child had a fever or ... thoroughly cooked, you would, of course, use a thermometer ... temperature within its range. However, it isn,t that simple ... systemstiny, channel-lined devices used in medical diagnostics, DNA forensics ...
... Since most parents in the US are employed, there ... choices for themselves and their children. How parents cope with ... choice coping strategies are the subjects of a study in ... and Behavior . Findings suggest that better work conditions may ...
... What about tree power? It turns out that it,s ... in trees for University of Washington researchers to run ... in an upcoming issue of the Institute of ... "As far as we know this is the first ...
Cached Biology News:NIST calculations may improve temperature measures for microfluidics 2Work conditions impact parents' food choices 2Electrical circuit runs entirely off power in trees 2Electrical circuit runs entirely off power in trees 3