NUCLEIC ACID FOR SUPPRESSING GENE EXPRESSION OF DISEASE-SPECIFIC COL3A1 MUTANT ALLELE AND PHARMACEUTICAL COMPOSITION FOR TREATING VASCULAR EHLERS-DANLOS SYNDROME

摘要

The present invention provides a pharmaceutical composition for use in treatment of a disease caused by a mutation in collagen 3A1. This pharmaceutical composition is for treating a disease caused by a collagen 3A1 mutant in a subject having a gene encoding the mutant and a gene encoding the wild type, said pharmaceutical composition containing a nucleic acid selected from siRNAs and shRNAs. The mutant is a gene encoding collagen 3A1 in which the base G at position 755 in the base sequence represented by SEQ ID No:27 is substituted with T or a gene encoding collagen 3A1 in which the base G at position 547 in the base sequence represented by SEQ ID No:27 is substituted with A. The nucleic acid suppresses the expression of the mutant more strongly than the expression of the wild type.