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:11/22/2014)... 2014 The “Chiral Chromatography Columns ... Material (Metal, Glass, Plastic), by Application [GC, LC ... Forecast to 2018” analyses and studies the major ... America, Europe, Asia, and Rest of the World. ... figures spread through 135 pages and in-depth TOC ...
(Date:11/22/2014)...  The element of surprise has been a major ... without warning. Until now, epidemiologists had nothing to measure ... resulting in suboptimal responses to both the H1N1 pandemic ... Photo - http://photos.prnewswire.com/prnh/20141121/160242 That ... virus gene structural changes have been found that invariably ...
(Date:11/21/2014)... November 21, 2014 , ...   Mariano Rodríguez es elegid vicepresidente senior ... KLOX está en marcha para comenzar de forma rápida ... heridas de reciente aprobación en Europa   , ... complace al anunciar los siguientes nombramientos: Todd ...
(Date:11/21/2014)... BELLINGHAM, Washington, USA (PRWEB) November 19, 2014 ... used in our lives was shared with more than ... undergraduate and postgraduate students from the University of Otago ... Outreach Grant . The Otago students, members of the ... Southland New Zealand in early September, giving children from ...
Breaking Biology Technology:Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 2Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 3Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 4Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 5Viruses' Advantage of Surprise is Lost 2KLOX 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 3
... Presented at the Pediatric Academic Societies Annual Meeting ... Statistically-Significant Association between SpCO and Measures of Lung ... /PRNewswire-FirstCall/ -- Masimo (Nasdaq: ... Measure-Through Motion and Low-Perfusion pulse oximetry, announced today ...
... Insmed Inc. (Nasdaq: INSM ), a biopharmaceutical company, today reported results ... , , ... Finalized sale of follow-on biologics (FOB) assets to Merck & Co., ... Net of finance and ...
... Pharmaceuticals, Inc. (Nasdaq: AMLN ) will be presenting ... Tuesday, May 19, 2009 at 11:30 a.m. ET / 8:30 ... chief executive officer of Amylin Pharmaceuticals, will provide a corporate ... a recording will be made available following the event. The ...
Cached Biology Technology:New Study Funded By the National Institutes of Health (NIH) Finds Masimo SpCO(R) May Provide a Noninvasive Measure of Acute Asthma Severity in Children 2New Study Funded By the National Institutes of Health (NIH) Finds Masimo SpCO(R) May Provide a Noninvasive Measure of Acute Asthma Severity in Children 3New Study Funded By the National Institutes of Health (NIH) Finds Masimo SpCO(R) May Provide a Noninvasive Measure of Acute Asthma Severity in Children 4New Study Funded By the National Institutes of Health (NIH) Finds Masimo SpCO(R) May Provide a Noninvasive Measure of Acute Asthma Severity in Children 5Insmed Announces First Quarter 2009 Financial Results 2Insmed Announces First Quarter 2009 Financial Results 3Insmed Announces First Quarter 2009 Financial Results 4Insmed Announces First Quarter 2009 Financial Results 5Insmed Announces First Quarter 2009 Financial Results 6Insmed Announces First Quarter 2009 Financial Results 7Insmed Announces First Quarter 2009 Financial Results 8Insmed Announces First Quarter 2009 Financial Results 9
(Date:11/4/2014)... Fla. (November 4, 2014) — Think about the way ... How do neighboring cells know that they are supposed ... how do these tissues find the correct place and ... answering these crucial questions. , In a new study, ... to communicate with their surrounding neighbors, at the head-trunk ...
(Date:11/4/2014)... , November 4, 2014   ... vertical market growth   Fuel3D , a ... closed a funding round totaling $6.4 million (£4 million). This ... funding secured earlier this year and paves the way for ... 2015. The funding round was led by Chimera ...
(Date:11/4/2014)... 2014   Neurotechnology , a provider of ... latest version of its fingerprint matching algorithm has ... evaluation organized by NIST. The MINEX test ... INCITS 378 fingerprint standard template format. MINEX compliance ... in the United States ...
Breaking Biology News(10 mins):The inside story: How the brain and skull stay together 2Fuel3D Secures $6.4 Million in Expansion Funding 2Fuel3D Secures $6.4 Million in Expansion Funding 3Neurotechnology Places Second in Ongoing MINEX Ranking for Fingerprint Matching Algorithms 2Neurotechnology Places Second in Ongoing MINEX Ranking for Fingerprint Matching Algorithms 3
... and the ECI Biochemical Engineering Conferences have announced ... at the University of Delaware and Director of the ... Biochemical Engineering Journal Young Investigator Award. This ... contributed to the field of biochemical engineering by a ...
... examining chromosomes in human eggs a few hours after ... a healthy baby, a researcher told the 25th annual ... Embryology today (Monday 29 June). Dr. Elpida Fragouli, from ... UK, and Reprogenetics UK, said that her team,s work ...
... Approximately 58 km southeast of Shingu City, Japan--Deepsea Drilling ... Nankai Trough Seismogenic Zone off the Kii Peninsula of ... in water depths of 2,054 meters. Following sea floor ... blow-out prevention (BOP) system into an upper section of ...
Cached Biology News:Kelvin Lee winner of Biochemical Engineering Journal Young Investigator Award 2New, less invasive genetic test greatly improves pregnancy rates in older women with poor prognosis 2New, less invasive genetic test greatly improves pregnancy rates in older women with poor prognosis 3First riser-drilling research operations undertaken 2
...
Mouse monoclonal antibody raised against a partial recombinant ZNF155. NCBI Entrez Gene ID = ZNF155...
Mouse polyclonal antibody raised against a partial recombinant COASY. NCBI Entrez Gene ID = 80347...
... Antibody against Nonmuscle Myosin II Heavy ... (Stored in TBS + 0.03% Thimerosal) ... peptide SDVNETQPPQSE corresponding to the C-terminus ... B (MHC-B) This antibody is effective ...
Biology Products: