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ASO corrects striatal transcriptional abnormalities & protects function in HD mice
Date:8/26/2013

ment on specific striatal-enriched transcripts in six-month old YAC128 and control mice. These results showed that "decreasing mutant HTT expression using an ASO directed specifically against human mutant HTT in YAC 128 mice significantly corrected the transcriptional profiles of DARPP32, enkephalin and CB1. D1 and D2 receptor levels also showed a trend towards improvement following ASO treatment," says Dr. Stanek.

The authors suggest that monitoring transcriptional changes could serve as a powerful tool for clinicians to follow HD progression and treatment. Since taking samples from human brain is not possible, alternative measures, such as changes in the content of mRNA or proteins in peripheral tissues or visualizing dysregulated receptors in brain using advanced neuroimaging techniques, may be developed as useful transcriptional biomarkers.


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Contact: Daphne Watrin
d.watrin@iospress.nl
31-206-883-355
IOS Press
Source:Eurekalert

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