Targeted Genetics Announces Breakthrough Research on Huntington's Disease

Targeted Genetics has announced that Beverly Davidson of the University of Iowa published encouraging preclinical results for a potential treatment of Huntington's disease (HD).

Davidson is an academic collaborator of Sirna Therapeutics, Targeted Genetics' partner for the development of novel therapies for the treatment of HD. The results of the independent preclinical study demonstrate that RNA interference (RNAi) therapy may have a beneficial impact on the symptoms and progression of HD.

The study appeared April 5 in the Online Early Edition of Proceedings of the National Academy of Sciences (PNAS) and will be published in the April 19 issue of PNAS.

With RNA interference therapy, researchers for the first time have been able to attack the fundamental cause of Huntington's disease and reduce the protein expression from the disease gene. The study is the first to show that a therapy designed to inhibit the expression of this protein has a beneficial effect on the disease symptoms. The study used RNAi to treat a mouse model of HD.

Results of the study demonstrated 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.