Navigation Links
The RNA drug revolution -- a new approach to gene therapy
Date:1/23/2008

New Rochelle, NY, January 23, 2008RNA interference (RNAi) represents an innovative new strategy for using small RNA molecules to silence specific genes associated with disease processes, and a series of review articles describing the state-of-the-art and potential therapeutic applications of RNAi and microRNAs will begin with two review papers in the January 2008 issue (Volume 19, Number 1) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The papers are available free online.

At least six clinical trials using RNA interference (RNAi) have been approved, with many more coming down the pipeline, according to the Editorial by Mark A. Kay, MD, PhD, an Associate Editor of Human Gene Therapy and the Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine. One thing is clear, adds Kay, small RNAs as a therapeutic platform are here to stay.

The excitement surrounding RNAi and the two main approaches to delivering RNA-based therapeuticsas mature siRNA molecules or as short hairpin RNAs (shRNAs)relates to the discovery of native microRNA molecules in human cells and their intrinsic ability to block the expression of a target gene. siRNA therapeutic strategies in development aim to harness the cells natural RNAi pathway and specifically silence a mutant or dysregulated gene.

Traditionally, gene therapy has focused on supplying a normal copy of a faulty gene, whereas RNAi turns off a problematic gene. These contrasting approaches share some of the same techniques and challenges, including delivery of a therapeutic gene or siRNA into cells.

Human Gene Therapy will publish a series of review papers in four consecutive issues focusing on a range of topics related to therapeutic applications of small RNAs. The series begins in the January issue with a paper entitled, Behind the Scenes of a Small RNA Gene-Silencing Pathway, by Gregory Ku, MD, PhD, and Michael McManus, PhD, from the University of California, San Francisco, which presents the current understanding of microRNA mechanisms of action and the potential for applying this knowledge to the development of RNAi-based treatments.

In the review by Anton McCaffrey, PhD, and Rebecca Marquez, MA, entitled, Advances in Micro-RNAs: Implications for Gene Therapists, the authors discuss the likelihood that microRNAs, which are believed to regulate as many as one-third of all human gene transcripts (or messenger RNAs), are implicated in many human diseases. Using gene therapy to manipulate microRNA levels represents an attractive new approach for controlling gene expression and identifying targeted and effective therapeutics.

"The concept of using RNA as a therapeutic product is quite attractive and adds an important new dimension to the field of nucleic acid based therapeutics including gene therapy. Mark Kay, Associate Editor of Human Gene Therapy, has organized an exciting series of reviews summarizing the state of the art of this emerging field," says James M. Wilson, MD, PhD, Editor-in-Chief, and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, in Philadelphia.


'/>"/>

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

Related medicine news :

1. Local Oncology Group Leads Online Effort to Revolutionize Cancer Care
2. Join the Vitamin D Revolution: How to Get 30 Minutes of Daily Sunshine for Optimal Health
3. VIDEO from Medialink and Revolution Health: RevolutionHealth.com Launches Resolutions 2.0
4. UCLA Program Aims to Revolutionize Kidney Transplants
5. Prescription for an electronic revolution?
6. Informed Medical Communications Sells Consumer Internet Division to Revolution Health
7. Chinas Anti-Aging Revolution
8. New Low-Impact Cosmetic Surgery Treatments Revolutionizing Industry
9. ILC Dover Launches Revolutionary ArmorFlex (R) Pharmaceutical Product Contact Film
10. The Homeopathic Revolution
11. Six Olympic Legends Battling Chronic Diseases Lead Revolution in Advancing Pharmacy Care as Medco Launches the Tour of Champions
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... ... October 13, 2017 , ... While it’s often important to ... Fortunately, an inventor from Austin, Texas, has identified a solution. , She developed a ... or restricted lighting. As such, it eliminates the need to turn on a light ...
(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... PurhealthRX ... CBD Oil utilizing Purzorb™ technology. Applying the Purzorb™process to full spectrum CBD oil will ... and providing a CBD form that can be easily incorporated into liquid products, while ...
(Date:10/13/2017)... ... 2017 , ... Apple Rehab Shelton Lakes , which specializes in the ... facility as part of a disaster drill on October 3rd. , Apple Rehab participated ... Emergency Manager, as well as the Connecticut Long Term Care Mutual Aid Plan ...
(Date:10/13/2017)... NY (PRWEB) , ... October 13, 2017 , ... Ellevate ... people in business to advocate for action towards gender equality at their inaugural Summit ... from around the globe, and reached a social audience of over 3 million. To ...
(Date:10/13/2017)... , ... October 13, 2017 , ... ... be giving viewers the lowdown on sciatica in a new episode of "Success ... focuses on current events and innovation and investigates each subject in-depth with passion ...
Breaking Medicine News(10 mins):
(Date:9/28/2017)... Cohen Veterans Bioscience and Early Signal Foundation announce a ... sensors for real-time monitoring of patients with trauma-related and ... focused on disruptive health solutions for rare disorders and ... record and integrate behavioral, cognitive, physiological and contextual data. ... ...
(Date:9/23/2017)... 22, 2017 Janssen Biotech, Inc. (Janssen) announced ... from the U.S. Food and Drug Administration (FDA) for ... for the treatment of moderately to severely active rheumatoid ... data are needed to further evaluate the safety of ... RA. "We ...
(Date:9/18/2017)... Mich. , Sept. 18, 2017  PMD Healthcare ... Specialty Pharmacy of Kalamazoo, Mich. , ... hub service that expedites and streamlines patient and provider ... PD 2.0, and wellness management services.  ... device used to measure lung function for a variety ...
Breaking Medicine Technology: