VIRAL VECTOR CARRYING SMN GENE EXPRESSION CASSETTE AND USE THEREOF

Abstract

Disclosed are an SMN gene expression cassette, a viral vector carrying the SMN gene expression cassette, and a use of the viral vector in treating spinal muscular atrophy, relating to the field of gene therapy. Codon optimization is carried out on an SMN1 gene in the gene expression cassette, so that the expression level of an SMN protein after SMN1 gene transduction is increased to effectively reduce the clinical dosage. Moreover, a regulatory element such as an miRNA target sequence is also introduced into the gene expression cassette to mitigate an immune response of an organism to an adeno-associated virus vector and a transgenic product, so as to reduce the safety problems possibly caused by gene therapy.

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