FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology. More specifically, the present invention relates to an isolated codon-optimized nucleic acid which codes SMN1 protein (survival protein of motor (motor) neurons), an expression cassette and a vector based thereon, as well as to recombinant virus based on AAV9 (adeno-associated serotype 9 virus) to increase expression of SMN1 gene in target cells, and use thereof.
EFFECT: disclosed is a codon-optimized nucleic acid which encodes a smn1 protein, and use thereof.
13 cl, 3 dwg, 1 ex
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