Retinal dystrophin transgene and methods of use thereof

摘要

Disclosed is a transformed vector comprising a nucleic acid sequence coding for human Dp260 protein; or a cell comprising a human Dp260 nucleic acid sequence inserted into the genome of cell and thereby transforming said cell; or a transgenic non-human animal having an exogenous human Dp260 nucleic acid sequence stably integrated into its genome. Further disclosed is the use of a genetic insert coding for human Dp260 protein in the manufacture of a medicament for reducing the severity of at least one clinical symptom of Duchenne Muscular Dystrophy (DMD) in an animal, wherein the genetic insert is introduced into the genome of said animal.