Navigation Links
Exon skipping prevents formation of toxic protein fragments in Huntington's disease
Date:2/11/2014

New Rochelle, NY, February 11, 2014An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin. Proof of concept using antisense oligonucleotides to "skip over" the specific exon in a mouse model of Huntington's disease is reported in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article, part of a special focus issue on exon skipping, is available on the Nucleic Acid Therapeutics website.

Melvin Evers et al., Leiden University Medical Center, The Netherlands, describe the successful use of antisense oligonucleotides to target the mutated exon that causes Huntington's disease in the article "Preventing Formation of Toxic N-Terminal Huntingtin Fragments Through Antisense Oligonucleotide-Mediated Protein Modification."

"No field of therapeutic development is moving faster, with more imminent clinical translation than the nucleic acid based treatment of central nervous system conditions," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI. "The novel therapeutic strategy outlined in Evers et al. gives us a proof of concept of how a previously intractable condition can be treated by modifying rather than removing the toxic protein."

The special issue also includes the Review article "A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications." by Peter Jrver, Liz O'Donovan, and Michael Gait, Medical Research Council, Cambridge, U.K. The authors explore the complex chemistry and design of antisense oligonucleotides used for exon skipping and progress in developing new chemistries to improve their stability and binding.

Annemieke Aartsma-Rus, PhD, Leiden University Medical Center, Guest Editor of the issue, emphasizes the need for scientists, clinicians, patients, regulators, and drug manufacturers to work closely together to develop exon skipping therapeutics, which are currently in clinical trials for neuromuscular disorders such as Duchenne muscular dystrophy and spinal muscular atrophy. These complex drugs and the challenging diseases they are targeting require a collaborative effort, she states in her Editorial "Antisense-Mediated Exon Skipping: Networking to Meet Opportunities and to Overcome Challenges."

Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Graham C. Parker, PhD.


'/>"/>

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

Related medicine news :

1. Skipping Meds May Raise Odds of ER Visits for Certain Medicare Patients
2. Many Americans Skipping Meds to Save Money, CDC Says
3. South Jersey Health and Body Explains the Best Ways to Get Nutrients in Your Body if The as Apposed to Always Skipping Meals
4. Skipping Aspirin Before Artery Procedure May Boost Death Rates
5. Weight-Loss Keys: Food Journals, Eating In, Not Skipping Meals
6. Physical fitness improves survival, prevents some heart attacks
7. Japanese superfood prevents flu infection
8. Get Rid of Herpes System Relieves Symptoms and Prevents Outbreaks
9. Avoiding specific region of brain during whole-brain radiotherapy prevents memory loss
10. Online USA Doctors/Virtual Doctors Creates Comprehensive Female Health Plan That Prevents Premature Death in Postmenopausal Women
11. Device Prevents Oil-Gas-/Well Fires Designed by InventHelp® Client (CCP-336)
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Exon skipping prevents formation of toxic protein fragments in Huntington's disease
(Date:6/23/2017)... ... June 23, 2017 , ... The Aesthetic Channel has recently ... Talei has come up with a proprietary technique that he calls the AuraLyft ... have dropped. For all ages, patients can expect to look refreshed, rejuvenated, and revitalized. ...
(Date:6/23/2017)... Georgia (PRWEB) , ... June 23, 2017 , ... ... Negative Breast Cancer Conference from Sept. 18 to 20. , The two-day conference ... (TNBC) subtype with the goal of improving patients’ lives and eliminating racial breast ...
(Date:6/23/2017)... Beach, FL (PRWEB) , ... June 23, 2017 , ... ... in Pinecrest, Florida. This is MD Now’s 28th facility overall and marks the urgent ... Dixie Highway, located one mile North of The Falls shopping mall. The new clinic ...
(Date:6/23/2017)... ... 2017 , ... Ross Insurance Agency ( http://www.rossinsuranceagency.com ) saves ... Agency’s (FEMA) recent update of flood zones, more people than ever are in ... enacted to reflect the actual risk in flood zone areas during a time ...
(Date:6/23/2017)... (PRWEB) , ... June 23, 2017 , ... Ulster University, ... June, 2017 from 9 am to 3 pm to present to graduate students exciting ... is an original curriculum project led by The Health Improvement Service of the ...
Breaking Medicine News(10 mins):
(Date:6/11/2017)... June 10, 2017  Eli Lilly and Company (NYSE: ... Phase 3 studies of galcanezumab, an investigational treatment for ... data on several key secondary endpoints for galcanezumab compared ... these studies (EVOLVE-1, EVOLVE-2 and REGAIN) will be presented ... meeting in Boston . ...
(Date:6/9/2017)... June 9, 2017 AirXpanders, Inc. (ASX: AXP) ... the design, manufacture, sale and distribution of the AeroForm® ... progress of its commercial roll-out in the ... more than one hundred (100) medical institutions and health ... offers a needle-free alternative for women who choose reconstructive ...
(Date:6/8/2017)...  Less than a month ago, amateur hackers executed ... including hospital networks, in over 150 countries. ... largest online extortion attempts ever recorded. With the increasing ... imperative that providers understand where the risks lie, and ... and many other very real cyber threats.  ...
Breaking Medicine Technology: