FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology. 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. Also described are compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to brain and/or spinal cord.
EFFECT: disclosed group of inventions can be used in medicine.
16 cl, 17 dwg, 4 ex
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