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TRANSGENIC RAT AND THE USE THEREOF IN THE ANIMAL MODEL FOR HUMAN HUNTINGDON'S DISEASE AND NUCLEAR CONSTRUCTS, VECTORS AND CELLS FOR THE PRODUCTION THEREOF
TRANSGENIC RAT AND THE USE THEREOF IN THE ANIMAL MODEL FOR HUMAN HUNTINGDON'S DISEASE AND NUCLEAR CONSTRUCTS, VECTORS AND CELLS FOR THE PRODUCTION THEREOF
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机译:转基因大鼠及其在动物模型中的应用(人类亨廷顿氏病和核构造,矢量和细胞)
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摘要
Huntingdon's Disease (HD) is an autosomal-dominant inherited progredient neurodegenerative disease from the group of CAG repeat/polyglutamine diseases and is characterized by a triad of psychiatric modifications, dementia and motory function disorders. On a sub-cellular level, a mutation with extended CAG trinucleotide repeats has been identified as the cause of Huntingdon's Disease. The therapeutic effects of certain substances can be tested on the neurochemically indicated transgenic animal models with expanded CAG repeats. In the present invention, transgenic rats were generated and characterized for human HD. Said rat model for human HD and other diseases of the CNS includes 51 CAG repeats under the control of a rat promoter and has a slowly progredient neurological phenotype closely reflecting human HD syndrome. The comparability of the rat model in relation to human HD is characterized by neuropathological, neuroradiological and neurochemical modifications accompanied by typical behavioural abnormalities.
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