Machado–Joseph disease (MJD) is a late-onset autosomal dominant neurodegenerative disorder, which is caused by a coding (CAG)n expansion in the ATXN3 gene (14q32.1). The number of CAG repeats in the expanded alleles accounts only for 50 to 75 % of onset variance, the remaining variation being dependent on other factors. Differential allelic expression of ATXN3 could contribute to the explanation of different ag...
An alternative approach to somatic gene therapy is to deliver the therapeutic protein by implanting genetically modified cells that could overexpress the gene of interest. Microencapsulation devices were designed to protect cells from host rejection and prevent the foreign cells from spreading while allowing protein secretion. Alginate microcapsules form a semi-permeable structure that is suitable for in vivo i...
Financiadores do RCAAP | |||||||
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