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US 2026/0306675 A1Published application
Epigenetic repression protein therapy for rasopathies
AssigneesThe Regents of the University of California, Battelle Memorial Institute
InventorsDavid J. SEGAL, Niraj PUNJYA, Kenneth R. SIMS, Jr., Chelsi SNOW
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Abstract
The present disclosure provides a CRISPR-based epigenetic repression system downregulating MAP2K1 and MAP2K2 expression for the treatment of RASopathies, such as neurofibromatosis type 1 (NF1).
Claims28
A recombinant polynucleotide comprising:
- (a) a first nucleic acid segment encoding a fusion protein comprising an RNA-guided programmable DNA-binding domain fused to a repressor domain;
- and
- (b) a second nucleic acid segment encoding at least one guide RNA targeting a MAP2K gene.
(canceled)
11-13. (canceled)
A system comprising:
- (a) a first polynucleotide encoding a fusion protein comprising an RNA-guided programmable DNA-binding domain fused to a repressor domain;
- and
- (b) a second polynucleotide encoding at least one guide RNA targeting a MAP2K gene.
(canceled)
24-25. (canceled)
A guide RNA targeting (i) the MAP2K1 gene having at least 80% identity to any one of SEQ ID NOS. 1-10, or (ii) the MAP2K2 gene having at least 80% identity to any one of SEQ ID NOS. 11-20.
27-29. (canceled)
(canceled)
Dependencies are inferred from the claim text.