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Oculopharyngeal muscular dystrophy: a paradigm to investigate new pharmaco-therapeutic approaches to trinucleotide-expansion diseases and muscular dystrophies


The goal of this proposal is to use oculopharyngeal muscular dystrophy (OPMD) as a paradigm to investigate new phannaco-therapeutic approaches based on genorne- and proteome-wide surveys of nonnal and dystrophic muscle. We will screen for genes/proteins capable of suppressing the dystrophic muscle phenotype. Selected candidates will represent the basis for developing a human gene therapy program. To reach this goal, we will generate cellular and animal models amenable to genetic screens and manipulations, and will construct DNA and protein microarrays to compare gene expression profiles in normal and dystrophic muscle. If a particular protein is found under-expressed in disease muscle we will isolate the corresponding cDNA and induce its over-expression in OPMD transgenic mouse model. Additional candidates for human gene therapy are expected to be identified by a genetic screen based on modification of the phenotype in the OPMD Drosophila model.

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Avenida Prof. Egas Moniz
1649-028 LISBOA

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Participants (5)