Effectiveness of using siRNA to treat Huntington’s disease

In Huntington’s disease, the mutant huntingtin gene (HTT) mRNA is retained in the nucleus and forms insoluble clusters. A new study shows that short interfering RNA (siRNA), an oligonucleotide therapeutic strategy that reduces levels of huntingtin mRNA in the cytoplasm, does not lower mutate HTT mRNA expression in the nucleus of mouse brains. The study is published in the peer-reviewed journal Nucleic Acid Therapeutics. Click here to read the article.

Sarah Allen, from the University of Massachusetts Chan Medical School, and coauthors, state: “Mutant mRNA forms nuclear clusters that are resistant to RNAi, while both nuclear and cytoplasmic wild-type Htt mRNA can be silenced. This study is the first to report on the impact of structure of nuclear RNA impacting efficiency of RNAi-based silencing.”

“Another key investigation from the laboratory of Anastasia Khvorova, to develop our understanding of the relationship between huntingtin RNA and protein levels where it matters functionally. says Executive Editor Graham C. Parker, PhD, Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

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. The Journal is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center and Annemieke Aartsma-Rus, PhD, Leiden University Medical Center, and Executive Editor Graham C. Parker, PhD. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a sample issue may be viewed on the Nucleic Acid Therapeutics website.

About the Society
The Oligonucleotide Therapeutics Society is an open, non-profit forum to foster academia- and industry-based research and development of oligonucleotide therapeutics. The society brings together the expertise from different angles of oligonucleotide research to create synergies and to bring the field of oligonucleotides to its full therapeutic potential.

About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.