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:11/21/2014)... , Todd Martensen se une ... Rodríguez es elegid vicepresidente senior de desarrollo empresarial y responsable ... para comenzar de forma rápida la comercialización de su sistema ... Europa   , KLOX Technologies Inc. ... nombramientos: Todd Martensen ha sido contratado ...
(Date:11/21/2014)... 19, 2014 The science of light ... shared with more than 200 primary students in a ... the University of Otago Physics Department and funded by ... students, members of the university’s OSA/SPIE Student Chapter visited ... September, giving children from small, rural schools hands-on lessons ...
(Date:11/21/2014)... CA (PRWEB) November 20, 2014 Author ... Short Walk to the Other Side” published as ... “Award-Winning Finalist in the “Fiction: Short Story” category of ... Jeffrey Keen, President and CEO of USA Book News, ... mainstream and independent publishers, including Simon & Schuster, Penguin, ...
(Date:11/21/2014)... The report, "Synthetic Biology Market by ... Technology (Bioinformatics, Nanotechnology, Gene Synthesis, Cloning & ... Global Forecast to 2018", analyses and studies ... challenges. , Browse 99 market data tables ... and in-depth TOC of the global synthetic ...
Breaking Biology Technology:KLOX Technologies anuncia sus nombramientos ejecutivos 2KLOX Technologies anuncia sus nombramientos ejecutivos 3KLOX Technologies anuncia sus nombramientos ejecutivos 4KLOX Technologies anuncia sus nombramientos ejecutivos 5Optics and photonics outreach programs share science, spark fascination with light 2Optics and photonics outreach programs share science, spark fascination with light 3Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 2Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 3Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 4Synthetic Biology Market Expected to Reach $5,630.4 Million in 2018 - New Report by MarketsandMarkets 2Synthetic Biology Market Expected to Reach $5,630.4 Million in 2018 - New Report by MarketsandMarkets 3Synthetic Biology Market Expected to Reach $5,630.4 Million in 2018 - New Report by MarketsandMarkets 4
... , , , PLEASANTON, ... Thoratec Corporation (Nasdaq: THOR ), a world leader ... restore failing hearts, and HeartWare International, Inc. (Nasdaq: ... heart pumps, announced today that the United States Federal Trade ...
... , RICHMOND, Calif., July 29 Sangamo ... 2009 financial results and accomplishments. , , ... consolidated net loss of $4.5 million, or $0.11 per share, compared ... share, for the same period in 2008. As of June 30, ...
... , VAN BUREN TWP., Mich. , ... of Ricardo plc, the leading independent provider of technology, product ... and new energy industries, has announced that the company is ... low carbon engine technologies. With the environmental demand to reduce ...
Cached Biology Technology:U.S. Federal Trade Commission to Challenge Thoratec's Proposed Acquisition of HeartWare International 2U.S. Federal Trade Commission to Challenge Thoratec's Proposed Acquisition of HeartWare International 3U.S. Federal Trade Commission to Challenge Thoratec's Proposed Acquisition of HeartWare International 4Sangamo BioSciences Reports Second Quarter 2009 Financial Results 2Sangamo BioSciences Reports Second Quarter 2009 Financial Results 3Sangamo BioSciences Reports Second Quarter 2009 Financial Results 4Sangamo BioSciences Reports Second Quarter 2009 Financial Results 5Sangamo BioSciences Reports Second Quarter 2009 Financial Results 6Sangamo BioSciences Reports Second Quarter 2009 Financial Results 7Ricardo Launches Consortium to Evaluate Lubricant Challenges of Future Low Carbon Engine Technologies 2Ricardo Launches Consortium to Evaluate Lubricant Challenges of Future Low Carbon Engine Technologies 3
(Date:11/11/2014)...  Forensicon, Inc., a Chicago -based ... the promotion of Yaniv Schiff from Senior ... Schiff,s new role as Director, he will lead company ... examiners and provide leadership within the company,s digital forensics ... Schiff joined Forensicon in 2006 ...
(Date:11/7/2014)... Researchers led by Dr. Debra Auguste, associate professor, ... at The City College of New York, have ... treatment for one of the most aggressive forms ... (TNBCs) have a high mortality rate owing to ... effective therapeutic options. However, Professor Auguste,s team, discovered ...
(Date:11/7/2014)... , November 6, 2014 Leading Biometric ... better mobile security revolutionizing online transactions.  Companies in focus today ... Ltd. (NYSE: BABA ), Google Inc. (NASDAQ: ... eBay Inc. (NASDAQ: EBAY ) and MasterCard Inc. ... and NXTDW), a biometric authentication company focused on the ...
Breaking Biology News(10 mins):Yaniv Schiff Promoted to Director of Digital Forensics at Forensicon, Inc. 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 3Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 4Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 5Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 6
... a light-triggered chemical tool, Johns Hopkins scientists report that they ... living cells and sending them to exact locations at precise ... greater command than ever in manipulating single molecules, allowing them ... cell behavior and to determine whether cells will grow, die, ...
... release is available in Spanish . ... growers find a replacement for methyl bromide are studying an ... ingredients. Researchers with USDA,s Agricultural Research Service (ARS) are ... microbial activity could be used to replace the popular fumigant. ...
... light on cells and then clicking off the light makes ... delivery system for chemotherapy drugs, as well as a non-invasive ... conclusion of a report in ACS,s The Journal of ... Aziz and colleagues note using this technique before to force ...
Cached Biology News:Hopkins researchers use light to move molecules 2Alternatives eyed for methyl bromide 2
Mouse polyclonal antibody to TNPO2 - transportin 2 (importin 3, karyopherin beta 2b)...
... HDAC4 - ChIP Grade HDAC4 is ... histone deacetylases, which consists of 1084 amino ... similar to the deacetylase domain of yeast ... the nucleus and cytoplasm in a process ...
...
Mouse monoclonal antibody to PDK2 - pyruvate dehydrogenase kinase, isoenzyme 2...
Biology Products: