By a News Reporter-Staff News Editor at Gene Therapy Weekly -- A new study on Genetics, Disorders and Diseases is now available. According to news reporting originating in Iowa City, Iowa, by NewsRx journalists, research stated, "Machado-Joseph disease (MJD) is a dominantly inherited ataxia caused by a polyglutamine-coding expansion in the ATXN3 gene. Suppressing expression of the toxic gene product represents a promising approach to therapy for MJD and other polyglutamine diseases."
The news reporters obtained a quote from the research from the University of Iowa, "We performed an extended therapeutic trial of RNA interference (RNAi) targeting ATXN3 in a mouse model expressing the full human disease gene and recapitulating key disease features. Adeno-associated virus (AAV) encoding a microRNA (miRNA)-like molecule, miRATXN3, was delivered bilaterally into the cerebellum of 6- to 8-week-old MJD mice, which were then followed up to end-stage disease to assess the safety and efficacy of anti-ATXN3 RNAi. Despite effective, lifelong suppression of ATXN3 in the cerebellum and the apparent safety of miRATXN3, motor impairment was not ameliorated in treated MJD mice and survival was not prolonged. These results with an otherwise effective RNAi agent suggest that targeting a large extent of the cerebellum alone may not be sufficient for effective human therapy."
According to the news reporters, the research concluded: "Artificial miRNAs or other nucleotide-based suppression strategies targeting ATXN3 more widely in the brain should be considered in future preclinical tests."
For more information on this research see: Toward RNAi Therapy for the Polyglutamine Disease Machado-Joseph Disease. Molecular Therapy, 2013;21(10):1898-1908. 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)
Our news correspondents report that additional information may be obtained by contacting M.D. Costa, University of Iowa, Dept. of Internal Med, Iowa City, IA 52242, United States. Additional authors for this research include K. Luna-Cancalon, S. Fischer, N.S. Ashraf, M. Ouyang, R.M. Dharia, L. Martin-Fishman, Y.M. Yang, V.G. Shakkottai, B.L. Davidson, E. Rodriguez-Lebron and H.L. Paulson (see also Genetics, Disorders and Diseases).
Keywords for this news article include: Iowa City, Iowa, United States, North and Central America, Bioengineering, Biotechnology, Brain Diseases, Brain Stem, Central Nervous System Diseases, Cerebellar Diseases, Cerebellum, Clinical Trials and Studies, Dyskinesias, Gene Therapy, Genetics, Disorders and Diseases, Machado-Joseph Disease, Metencephalon, Neurodegenerative Diseases, Neurologic Manifestations, Pre-Trial Research, Spinal Cord Diseases, Spinocerebellar Ataxias, Spinocerebellar Degenerations, Therapeutics
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