Method for treating muscular dystrophy by targeting LAMA1 gene
AssigneeModalis Therapeutics Corporation
InventorsYuanbo Qin, Tetsuya Yamagata
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Abstract
The present invention aims to provide a novel therapeutic approach to human muscular dystrophy (particularly MDC1A). The present invention provide 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 activator, and (b) a base sequence encoding (i) a guide RNA targeting a continuous region set forth in SEQ ID NO: 15, 20, 25, 50, 56, or 61, (ii) a guide RNA targeting a continuous region set forth in SEQ ID NO: 124, or (iii) a guide RNA targeting a continuous region set forth in SEQ ID NO: 178, 193, or 195, in the expression regulatory region of human LAMA1 gene.
Claims13
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 activator, and
- (b) a base sequence encoding a guide RNA:
- (i) hybridizing with one of SEQ ID NO:
- 15, 20, 25, 50, 56, or 61, or
- (ii) hybridizing with SEQ ID NO:
- 124, or
- (iii) hybridizing with one of SEQ ID NO:
- 178, 193, or 195, in the expression regulatory region of human LAMA1 gene;
- wherein the nuclease-deficient CRISPR effector protein is dCas9;
- and
- wherein the dCas9 is derived from Staphylococcus aureus.
Dependencies are inferred from the claim text.