FIELD: biotechnology.
SUBSTANCE: invention relates to a genetic construct carrying an open reading frame of the human SMN1 gene, controlled by a neurospecific promoter of the synapsin I (SYN1) gene, and can be used in medicine. Due to the SYN1 promoter contained in the vector, transgene expression will be ensured only in neuronal cells, and the presence of inverted AAV-ITR terminal repeats ensures packaging of the recombinant adeno-associated virus into virions.
EFFECT: invention can be used in the production of a gene therapy medicinal product for combating spinal muscular atrophy type 1 (SMA1) by delivering a construct using a recombinant adeno-associated virus of serotype 2.
1 cl, 1 dwg
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