| 摘要 |
PROBLEM TO BE SOLVED: To provide 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.SOLUTION: The invention relates to a self-complementary (SC) adeno-associated virus (AAV) vector for treating motor neuron disorders, such as spinal muscular atrophy, amyotrophic lateral sclerosis, spinobulbar muscular atrophy, spinal cerebellar ataxia, primary lateral sclerosis, and traumatic spinal cord injury. A recombinant AAV virion in which the viral vector comprises a nucleic acid encoding a survival neuron disorder (SMN)-1 protein of a survival motor neuron; and a method for use thereof are also provided.SELECTED DRAWING: None |
