Research paper on innovative use of RNAi technology described as “Top Methods” paper by Nucleic Acids Research

December 26, 2003

WORCESTER, Mass.— An innovative paper on the use of “short hairpin” RNAi technology by University of Massachusetts Medical School faculty has been cited as a “Top Methods” paper by the scientific journal Nucleic Acids Research.  The paper, “An enhanced U6 promoter for synthesis of short hairpin RNA” by UMMS Associate Professor of Biochemistry & Molecular Pharmacology Zuoshang Xu, PhD, and colleagues, appeared in the September 1, 2003 issue of Nucleic Acids Research, an Oxford online journal that publishes the results of leading edge research on nucleic acids and proteins involved in nucleic acid metabolism and/or interactions.  The online journal enables the rapid publication of papers under the following categories: RNA, molecular biology, chemistry, genomics, computational biology and structural biology, and in 2002 was in the top 25 scientific journals for scientific impact, as measured by the frequency with which papers published in the journal are cited by other researchers.

The publication details the identification of a method that enhances the recently discovered genetic process known as “RNA interference” (RNAi) in the study of the degenerative neurological disease amyotrophic lateral sclerosis (ALS, commonly referred to as  Lou Gehrig’s Disease); the method described by Xu and colleagues may be an important tool for researchers studying the important effects of RNAi and its role in the process of silencing the activity of genes.

The study of ALS at the University of Massachusetts Medical School is supported by a sponsored research agreement with the biotech company CytRx Corporation, which has an exclusive, worldwide license agreement with UMMS to use RNAi gene silencing technology in the development of a treatment for  ALS.   According to Dr. Xu, RNAi has the potential to help determine the underlying cause of ALS at the genetic level by searching for and selectively silencing the mutant, disease-causing, gene.  The NAR paper, by identifying an enhanced promoter for a key RNA transcription step, may help researchers more easily determine the most advantageous therapeutic strategy for ALS.

Dr. Xu said, “RNAi technology has a great potential for treatment of many genetic as well as non-genetic diseases. If we can understand the fundamentals of RNAi and successfully apply it to treat ALS, it will not only benefit the ALS patients but also open doors for treatment of other diseases.”

“This important publication is a good example of the potential of RNAi technology in the fight against ALS," said John L. Sullivan, MD, Director of the Office of Research at the University of Massachusetts Medical School. “Like many of his UMMS colleagues, Dr. Xu is working at the leading edge of development of this promising new field of biomedical science.”

The publication may be found at

ALS was first described in scientific literature in 1869, and there is still no cure. The exact cause of this progressive neurodegenerative disease that results in motor neuron degeneration of the brain and spinal cord and eventual paralysis is unknown.  In 50% of patients, death normally occurs 18 months after diagnosis. Only 20% of patients survive five years and 10% live longer than ten years. According to the ALS Association, in the United States alone, approximately 30,000 people are living with ALS and almost 15 new cases are diagnosed daily.

The University of Massachusetts Medical School, one of the fastest growing academic health centers in the country, has built a reputation as a world-class research institution, consistently producing noteworthy advances in clinical and basic research.  The Medical School attracts more than $151 million in research funding annually, 80 percent of which comes from federal funding sources.  Research funding enables UMMS scientists to explore human disease from the molecular level to large-scale clinical trials.  Basic and clinical research leads to new approaches for diagnosis, treatment and prevention of disease. 


Contact: Mark L. Shelton 508-856-2000