Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease.
作者： Shilpa Ramaswamy , Jodi L. McBride , Ina Han , Elizabeth M. Berry-Kravis , Lili Zhou
DOI: 10.1016/J.NBD.2008.12.005
关键词:
摘要: Members of the GDNF family ligands, including neurturin (NTN), have been implicated as potential therapeutic agents for Huntington's disease (HD). The present study examined ability CERE-120 (AAV2-NTN) to provide structural and functional protection in N171-82Q transgenic HD mouse model. AAV2-NTN therapy attenuated rotorod deficits this mutant relative control treated transgenics (p<0.01). treatment significantly reduced number mice that exhibited clasping behavior partially restored their stride lengths (both p<0.05). Stereological counts NeuN-ir neurons revealed a significant neuroprotection striatum (p<0.001). Most fascinating, stereological NeuN-labeled cells layers V-VI prefrontal cortex intrastriatal administration prevented loss frontal cortical seen These data indicate gene delivery NTN may be viable strategy incurable disease.
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