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:10/13/2017)... ... 2017 , ... The International Association of Eating Disorders Professionals ... the field of eating disorders, announces the opening of early registration for the ... at the Omni Resort at ChampionsGate. , The annual iaedp™ Symposium ...
(Date:10/13/2017)... ... ... Apple Rehab Shelton Lakes , which specializes in the delivery of ... part of a disaster drill on October 3rd. , Apple Rehab participated with the ... as well as the Connecticut Long Term Care Mutual Aid Plan (LTC-MAP). The ...
(Date:10/13/2017)... Milford, NJ (PRWEB) , ... October 13, 2017 , ... ... weekend at scenic Alexandria Park in Milford, NJ. This free event, sponsored by ... and physical activity. The fun run is geared towards children of all ages; ...
(Date:10/13/2017)... ... ... “The Journey: From the Mountains to the Mission Field”: the story of a missionary ... Journey: From the Mountains to the Mission Field” is the creation of published author, ... ages and currently teaches a class of ladies at her church, which she has ...
(Date:10/12/2017)... , ... October 12, 2017 , ... Planet Fitness, one ... U.S., announced today its plans to open a flagship location in Covington, LA at ... former Rooms To Go store next to Office Depot in the Holiday Square shopping ...
Breaking Medicine News(10 mins):
(Date:9/19/2017)... ANN ARBOR, Mich. , Sept. 19, 2017 HistoSonics, Inc., a venture-backed medical ... the precise destruction of targeted tissues, announced three leadership team developments today:   ... Josh Stopek, PhD ... ... Veteran medical device executive ...
(Date:9/18/2017)... KALAMAZOO, Mich. , Sept. 18, 2017 /PRNewswire/ ... and OptiMed Specialty Pharmacy of Kalamazoo, Mich. ... a strategic hub service that expedites and streamlines patient ... spirometer, Spiro PD 2.0, and wellness management services.  ... a medical device used to measure lung function for ...
(Date:9/13/2017)... , Sept. 13, 2017   OrthoAtlanta has ... the Atlanta Football Host Committee (AFHC) for the 2018 College ... Jan. 8, 2018, at Mercedes-Benz Stadium in Atlanta, ... of the AFHC "I,m In" campaign, participating in many activities ... ...
Breaking Medicine Technology: