Oligomeric nucleic acid molecules and their use for the treatment of acute intermittent porphyria

摘要

The present invention relates to compositions of small activating nucleic acid molecules that increase the expression of HMBS genes and their use. The small activating nucleic acid molecule may 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 region of complementarity, and the region of complementarity may form a double-stranded nucleic acid structure capable of promoting expression of the HMBS gene. The first nucleic acid strand or the second nucleic acid strand independently has a length of 16 to 35 nucleotides. The 3' end of the two oligonucleotide strands may have an overhang of 0 to 6 nucleotides in length. A small activating nucleic acid molecule for the HMBS gene can be used to up-regulate mRNA and protein expression of the HMBS gene and promote its enzymatic activity.