Treatment of neurodegenerative diseases by targeting miRNA

摘要

The present invention relates to a pharmaceutical composition for preventing or treating neurodegenerative diseases by targeting a specific miRNA. In addition, the present invention relates to a kit for diagnosing neurodegenerative diseases. A miR-206 target found in the present invention, which is highly expressed in both animal models of Alzheimer's disease and human brain samples, is a substantial treatment target selected without artifact errors. An antisense oligonucleotide of the present invention as an inhibitor for miR-206 suggests a successful result in treatment of neurodegenerative diseases by targeting miRNA. The antisense oligonucleotide of the present invention inhibits the function of miR-206 to greatly increase the levels of BDNF and IGF-1 and to increase the regeneration of synapses, thereby treating neurodegenerative diseases, particularly Alzheimer's disease.

主权项

1. A method for treating an Alzheimer's disease, comprising administering to a subject in need thereof a pharmaceutical composition comprising:
(a) a pharmaceutically effective amount of an antisense oligonucleotide having perfect complementarity to SEQ ID NO:1; and (b) a pharmaceutically acceptable carrier, wherein the antisense oligonucleotide is a 2′—O—C1-3 alkyl ribonucleotide.