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:8/23/2017)... ... 23, 2017 , ... PreCheck, Inc., a background screening and ... with a three-year sales growth of 49 percent . The Inc. 5000 is ... comprehensive look at America’s independent entrepreneurs. This represents PreCheck’s fifth consecutive entry on ...
(Date:8/23/2017)... Lauderdale, FL (PRWEB) , ... August 23, 2017 ... ... firms for traveling medical professionals, recently launched a new website, http://www.cirrusmedicalstaffing.com . ... built to respond to the needs of healthcare professionals who are traveling on ...
(Date:8/23/2017)... ... August 23, 2017 , ... Arizona Spine & Joint Hospital ... the designation of High Performing Hospitals by U.S. News & World Report for knee ... was significantly better than the national average in a given procedure or condition.  ...
(Date:8/23/2017)... ... August 23, 2017 , ... Boling Vision Center is excited ... and is now seeing patients at Boling Vision Center’s Elkhart & Notre Dame ... and treatment of eye disease, medical retina, high-technology cataract surgery, cosmetic procedures, and ...
(Date:8/23/2017)... OH (PRWEB) , ... August 23, 2017 , ... ... accessible vans, announced today that has been named to the 2017 Inc. 500|5000, ... 2545 based on a three-year growth rate of 139 percent, marking the twelfth ...
Breaking Medicine News(10 mins):
(Date:8/18/2017)... 2017  Nortis today announced the awarding of a $688K National Institutes of ... year of a Small Business Innovation Research (SBIR)?Fast-track grant under ... Disorders and Stroke (NINDS).?  ... Nortis ParVivo Chip used for 3D ... The neurotherapeutics ...
(Date:8/17/2017)... Aug. 17, 2017 MJAC2017 , the premier ... Broadcast Media, today reveals its leading lineup. ... a global cannabis innovator specializing in the development of pharmaceutical ... conference. MassRoots, Rambridge™ and The Green Organic Dutchman are gold ... Namaste Vapes™ as silver sponsors. ...
(Date:8/17/2017)... 2017 DarioHealth Corp. (NASDAQ: DRIO), a ... and big data solutions, announced today that it ... non-U.S. investors for the sale of shares of ... Company,s newly designated Series B Convertible Preferred Stock ... to conduct a closing with respect to the ...
Breaking Medicine Technology: