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Follow on Google News | Exon Skipping Prevents Formation of Toxic Protein Fragments in Huntington's DiseaseExon Skipping Prevents Formation of Toxic Protein Fragments in Huntington's Disease New Rochelle, NY—An 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 (http://www.liebertpub.com/ 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- “No field of therapeutic development is moving faster, with more imminent clinical translation than the nucleic acid based treatment of central nervous system conditions," The special issue also includes the Review article "A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications," 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- About the Journal Nucleic Acid Therapeutics is an authoritative, peer-reviewed journal published bimonthly in print and online that focuses on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a free sample issue may be viewed on the Nucleic Acid Therapeutics website. About the Society The Oligonucleotide Therapeutics Society (http://www.oligotherapeutics.org) About the Publisher Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many areas of science and biomedical research, including Human Gene Therapy, Human Gene Therapy Methods, Human Gene Therapy Clinical Development, ASSAY and Drug Development Technologies, and DNA and Cell Biology. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website (http://www.liebertpub.com) Contact vcohn@liebertpub.com End
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