Genetically Engineered Mouse Models of Neurodegenerative Disorders

摘要: Considerable advances have been made in the past years developing novel experimental models of neurodegenerative disorders, including Alzheimer’s disease (AD), Parkinson’s (PD), Fronto-Temporal Dementias (FTD), Amyotrophic Lateral Sclerosis (ALS), and trinucleotide repeat disorders (TNRDs). The main postulate several genetically modified murine is that a single molecular alteration might trigger cascade events eventually will result full spectrum clinicopathological alterations observed human disease. Therefore, overexpression mutant proteins transgenic (tg) mice mimic some aspects associated with gain toxic function, while targeted deletion selected genes loss trophic or protective function. To date, identified to be familial forms AD, PD, FTD, ALS, TNRDs. Single combined tg knockout targeting most these developed recapitulate one each disorder. In models, abnormal accumulation misfolding (toxic conversion) endogenous being extensively explored as key pathogenic event leading neurodegeneration. Thus, focus this chapter provide perspective efforts engineered common disorders. Further development investigation animal diseases holds promise better understanding their pathogenesis discovering testing new treatments.