FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a self-complementary adeno-associated viral (scAAV) vector for modulation of motor function in a subject, recombinant AAV virion, composition, containing said AAV virion, a method for modulating motor function in a subject with motor neuron disturbance, a method for providing an SMN protein to a subject, use of recombinant AAV virion in preparing a drug for modulating motor function in a subject with motor neuron disturbance and use of the recombinant AAV virion in the manufacture of a drug for providing SMN protein cells of a subject suffering spinal muscular atrophy (SMA). In one embodiment, the self-complement adeno-associated viral (scAAV) vector comprises a polynucleotide encoding a capsid from AAV serotype 9, and a polynucleotide encoding a motor neuron survival protein (SMN).
EFFECT: invention extends the range of agents for modulating motor function in a subject.
16 cl, 4 ex, 17 dwg
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