METHOD FOR TREATING ALZHEIMER'S DISEASE BY TARGETING MAPT GENE

摘要

A polynucleotide, comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcription repressor, and (b) a base sequence encoding a guide RNA targeting a continuous region of 18 to 24 nucleotides in length in a region set forth in SEQ ID NO: 54, 55, 56, 57, 68, 153 or 97 in the expression regulatory region of human MAPT gene. are expected to be useful for treating or preventing tauopathy including Alzheimer's disease.