Development Status
Dr. Beverly Davidson at the University of Iowa has published preclinical data demonstrating that the delivery of siRNA to the brain using an AAV vector efficiently inhibited gene expression in an animal model of spinocerebellar ataxia 1 (SCA1), a member of a class of inherited human neurodegenerative diseases that includes Huntington's disease (Nature Medicine, 2004). Dr. Davidson also has conducted studies in which AAV vectors were used to express siRNAs and were directly injected into the brain of mice with HD. Results of these studies demonstrate nearly normal movement in the mice and significant improvements in characteristic neurological damage compared to untreated mice. The study also demonstrated that levels of toxic HD protein in siRNA-treated mice were reduced to 40 percent of normal levels (PNAS, 2005).
Preclinical studies in small and large animal models are ongoing in the laboratory of Dr. Beverly Davidson.
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