Sirna Therapeutics has announced the publication of preclinical studies evaluating RNA interference-based therapies for a class of inherited neurodegenerative diseases of the central nervous system (CNS) in the current issue of Nature Medicine.
In a study led by Dr. Beverly Davidson, Roy J. Carver Chair in Internal Medicine at the University of Iowa, expression of a gene responsible for a disease mimicking the human neurodegenerative disease spinocerebellar ataxia 1 (SCA1) was efficiently inhibited by RNA interference technology in a mouse model of the disease. SCA1, like Huntington's Disease, is a member of a family of devastating neurological diseases known as polyglutamine diseases that strike most often in midlife, causing progressive and unrelenting physical and mental deterioration.
Mice with the SCA1 disease gene treated with RNAi-based compounds had normal movement and coordination. The RNAi treatment also protected brain cells from the destruction normally caused by the disease and prevented the build-up of protein clumps within the cells. In contrast, untreated mice developed movement problems and lost brain cells in a manner similar to humans with this condition. The study, conducted by scientists at the University of Iowa College of Medicine and colleagues at the University of Minnesota and the National Institutes of Health (NIH), appeared in the August issue of Nature Medicine (Volume 10, pp 816, August 2004). This seminal work of Dr. Davidson and her colleagues will be important for the future development of RNAi-based therapeutics against this and other related neurological conditions such as Huntington's, Parkinson's, and Alzheimer's Diseases.