Riboswitches for regulating gene expression and therapeutic methods of using the same

Abstract

The present disclosure provides an expression cassette comprising from 5' to 3': (i) promoter, (ii) an upstream exon, (iii) an upstream intron, (iv) an alternatively spliced exon comprising a translation initiation sequence, (v) a downstream intron, (vi) a downstream exon, and (vii) a heterologous nucleic acid sequence. In some aspects, the upstream exon, the upstream intron, the alternatively spliced exon, the downstream intron, and the downstream exon, each comprise a portion of a gene selected from the group consisting of a Transient Receptor Potential Cation Channel Subfamily V Member 3 (TRPV3) gene, a Serine / Threonine Kinase 31 (STK31) gene, an Ubiquitin Specific Peptidase 25 (USP25) gene, a IK gene, a Testis Expressed 14, Intercellular Bridge Forming Factor (TEX14) gene, a General Transcription Factor IIB (GTF2B) gene, and a LY6 / PLAUR Domain Containing 3 (LYPD3) gene.

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