Treatment of facioscapulohumeral muscular dystrophy targeting the DUX4 gene
JP7736329B2Active Publication Date: 2025-09-09MODALIS THERAPEUTICS CORP
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Patent Information
- Application Number
- JP2023513722
- Authority / Receiving Office
- JP · JP
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2021-08-20
- Filing Date
- 2021-08-31
- Publication Date
- 2025-09-09
- Estimated Expiration
- 2041-08-31
Abstract
A polynucleotide comprising the following base sequence: (a) a nucleotide sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcriptional repressor; and (b) A base sequence encoding a guide RNA targeting a consecutive region represented by SEQ ID NO: 2, 3, 4, 20, 51, 68, 144, 148, 152, 162, 164, or 167 in the expression control region of the human DUX4 gene is expected to be useful for the treatment or prevention of facioscapulohumeral muscular dystrophy (FSHD).
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Citation Information
Patent Citations
Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2013176772A1
Silencing of DUX4 by recombinant gene editing complexes
WO2018057863A1