公开(公告)号：EP2424991A1

公开(公告)日：2012-03-07

申请号：EP10772363.7

申请日：2010-04-27

申请人： Genzyme Corporation

发明人： PASSINI, Marco, A. ,  SHIHABUDDIN, Lamya ,  CHENG, Seng, H.

IPC分类号： C12N15/79 ,  A61K31/7088

CPC分类号： A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed. Also disclosed is a self-complementary adeno-associated virus vector for treating motor neuron disorders such as spinal muscular atrophy, amytrophic lateral sclerosis, spinal bulbar muscular atrophy, spinal cerebellar ataxia, primary lateral sclerosis, and traumatic spinal cord injury.

摘要翻译： 公开了用于治疗影响运动功能的疾病的组合物和方法，所述运动功能诸如受疾病或脑和/或脊髓损伤影响的运动功能。 还公开了用于治疗运动神经元病症例如脊髓性肌萎缩症，萎缩性侧索硬化症，脊髓延髓肌萎缩症，脊髓小脑性共济失调症，原发性侧索硬化症和创伤性脊髓损伤的自我补充腺相关病毒载体。