Oligomeric nucleic acid molecule, and application thereof in an acute intermittent porphyria treatment

Abstract

The present invention relates to compositions of small activating nucleic acid molecules for increasing the expression of HMBS gene and a use thereof. The small activating nucleic acid molecule can be a double-stranded or single-stranded RNA molecule targeting the promoter region of the HMBS gene. The first nucleic acid strand and the second nucleic acid strand each contain a complementary region, and the complementary regions can form a double-stranded nucleic acid structure, which can promote the expression of the HMBS gene. The first nucleic acid strand or the second nucleic acid strand independently have a length of 16 to 35 nucleotides. The 3′ terminus of the two oligonucleotide strands may have an overhang of 0 to 6 nucleotides in length. The small activating nucleic acid molecule for the HMBS gene can be used to up-regulate mRNA and protein expressions of the HMBS gene in a cell and promote enzymatic activity thereof

Dependencies are inferred from the claim text.