Genetically modified animal models have been extensively used to investigate the pathogenesis of age-dependent neurodegenerative diseases,such as Alzheimer(AD),Parkinson(PD),Huntington(HD) diseases,and Amyotrophic lat...
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Genetically modified animal models have been extensively used to investigate the pathogenesis of age-dependent neurodegenerative diseases,such as Alzheimer(AD),Parkinson(PD),Huntington(HD) diseases,and Amyotrophic lateral sclerosis(ALS). The common feature of these diseases is the age-dependent accumulation of misfolded proteins in the brain,which can be recapitulated in a variety of mouse models of neurodegenerative diseases. However,the brains of transgenic mouse models of AD,PD,and HD do not show the striking neuronal loss or degeneration that is a typical pathological feature in patient brains. Species differences between small animals and humans may account for differential pathology in transgenic mouse models and patient brains. Using CRISPR/Cas9 to modify the endogenous disease genes in large animals(pigs and monkeys),we demonstrate that typical neuropathological features can be mimicked in the brains of large animals. The findings underscore the importance of using large mammals to investigate the pathogenesis of important brain diseases and their therapeutics.
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