【摘 要】
：
Huntingtons disease (HD) is a well-characterized disorder that shares many common pathological features,such as age-de-pendent accumulation of misfolded proteins and neuronal degeneration,with other i
【机 构】
：
State Key Laboratory of Molecular Developmental Biology, Institute of Genetics and Developmental Bio
【出 处】
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Joint Symposium of 2016 International Neural Regeneration Sy
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Huntingtons disease (HD) is a well-characterized disorder that shares many common pathological features,such as age-de-pendent accumulation of misfolded proteins and neuronal degeneration,with other important neurodegenerative diseases including Alzheimer and Parkdnson diseases.Thus,elucidating the pathogenesis of HD is also important for understanding and treatment of other age-dependent neurodegenerative diseases.Although transgenic mouse models of HD have been widely used and provided important insight into the HD pathogenesis,however,they do not develop overt neuron apopto-sis in the brain,an important pathological hallmark in the brain of HD patients.Also,many drugs that are effective to treat HD mice fail to produce the similar therapeutic effects in humans.The brain circuitry in pigs and monkeys is more complex than in mice and they are ploy gyria.The previous studies have established transgenic HD pig and monkey models,but these transgenic animals were unable to survive due to the overexpression of the toxic form protein and different copy numbers and locus site of mutant huntingtin.Using CRISPR/Cas9 and nuclear transfer technologies,we have obtained newborn pigs that carry the HD mutation in the endogenous pig Huntingtin gene.These pigs showed the expression of full-length mutant huntingtin at the endogenous level.Using these Huntingtin knock-in pigs,we will examine their behaviors,motor function,and growth.These studies aim to identify important pathogenic evidence in Huntingtin knock-in pigs,which will help us to develop more effective therapeutic strategies for HD and other neurodegenerative diseases.
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