By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Researchers detail new data in Central Nervous System. According to news originating from Iowa City, Iowa, by NewsRx correspondents, research stated, "Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disease caused by a polyglutamine expansion in the deubiquitinating enzyme, Ataxin-3. Currently, there are no effective treatments for this fatal disorder but studies support the hypothesis that reducing mutant Ataxin-3 protein levels might reverse or halt the progression of disease in SCA3."
Our news journalists obtained a quote from the research from the University of Iowa, "Here, we sought to modulate ATXN3 expression in vivo using RNA interference. We developed artificial microRNA mimics targeting the 3'-untranslated region (3'UTR) of human ATXN3 and then used recombinant adeno-associated virus to deliver them to the cerebellum of transgenic mice expressing the full human disease gene (SCA3/MJD84.2 mice). Anti-ATXN3 microRNA mimics effectively suppressed human ATXN3 expression in SCA3/MJD84.2 mice. Short-term treatment cleared the abnormal nuclear accumulation of mutant Ataxin-3 throughout the transduced SCA3/MJD84.2 cerebellum. Analysis also revealed changes in the steady-state levels of specific microRNAs in the cerebellum of SCA3/MJD84.2 mice, a previously uncharacterized molecular phenotype of SCA3 that appears to be dependent on mutant Ataxin-3 expression."
According to the news editors, the research concluded: "Our findings support the preclinical development of molecular therapies aimed at halting the expression of ATXN3 as a viable approach to SCA3 and point to microRNA deregulation as a potential surrogate marker of SCA3 pathogenesis."
For more information on this research see: Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice. Molecular Therapy, 2013;21(10):1909-1918. Molecular Therapy can be contacted at: Nature Publishing Group, 75 Varick St, 9TH Flr, New York, NY 10013-1917, USA. (Elsevier - www.elsevier.com; Molecular Therapy - www.elsevier.com/wps/product/cws_home/622922)
The news correspondents report that additional information may be obtained from E. Rodriguez-Lebron, University of Iowa, Dept. of Physiol & Biophys, Iowa City, IA 52242, United States. Additional authors for this research include M.D. Costa, K. Luna-Cancalon, T.M. Peron, S. Fischer, R.L. Boudreau, B.L. Davidson and H.L. Paulson (see also Central Nervous System).
Keywords for this news article include: Biotechnology, Iowa City, Brain Stem, Cerebellum, Gene Therapy, United States, Metencephalon, Bioengineering, Central Nervous System, North and Central America
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