drosophila transgenic animal models for myotonic dystrophies.

摘要

Transgenic animals in Drosophila models for myotonic dystrophies.; The invention relates to the application of a transgene includes totally or partially, the gene sequence muscleblind, Drosophila or their counterparts in humans (eg MBNL), to a transgenic animal model. This transgenic model has been obtained by stable integration of the transgene into the genome of Drosophila melanogaster. In these animals, the transgene is expressed phenotypically, controlled manner, in any of its stages of development. Animal models of the invention are applicable in the investigation of the mechanisms of pathogenesis responsible for myotonic dystrophies as assay devices potential therapeutic compounds for the treatment of such diseases and as validators in vivo potential treatments thereof .