Kurzfassung

Gene therapy is a newly emerging field with promising treatments of the most complex mammalian organ, the brain. Efficacy and safety in the treatment of brain disorders depend on the ability to target the expression of therapeutic genes exclusively to those cells affected by a genetic defect. Our aim is to identify and characterize novel genetic tools allowing for transfer of curative genetic material exclusively in those cells that are dysfunctional in white matter diseases. More specifally,...Gene therapy is a newly emerging field with promising treatments of the most complex mammalian organ, the brain. Efficacy and safety in the treatment of brain disorders depend on the ability to target the expression of therapeutic genes exclusively to those cells affected by a genetic defect. Our aim is to identify and characterize novel genetic tools allowing for transfer of curative genetic material exclusively in those cells that are dysfunctional in white matter diseases. More specifally, we propose to combat the lethal childhood brain disorder Canavan disease by defining novel strategies to replace the defective gene underlying the disease in an existing animal model. This project will help to define ways to ameliorate the disease, but also to better define the underlying pathomechanism. If successful, findings are likely to be translated in clinical trials for this lethal condition for which there is no medical treatment currently available. Our proposed experiments include virally-mediated gene transfer to rodents, and results obtained will be applicable to the development of genetic treatments for other neurological conditions such multiple sclerosis (MS).» weiterlesen» einklappen