Animal models for cell and gene therapy in myelin disease

Disorders of myelination are multifaceted and often complex. Animal models cannot, in most cases, mimic the entire complexity of these diseases. This, however, may be neither necessary nor always helpful. Rather, the choice of an appropriate animal model should be based on the pathogenic facet to be studied and the therapeutic approach to be evaluated for tackling a specific disease component. Based on this hypothesis, this review examines the impact of various animal models on the preclinical validation of novel cell and gene therapy strategies for the treatment of myelin disease.

Section editor:Wolfgang Wurst – Institute of Developmental Genetics, GSF Research Centre, Neuherberg, Germany