Methods and compositions for treating urea cycle disorders, in particular otc deficiency
AssigneeCamp4 Therapeutics Corporation
InventorsAlfica Sehgal, Alla A. Sigova, Igor Zlobine, Brian E. Schwartz, David A. Bumcrot
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Abstract
The present invention provides methods and compositions for the treating a patient with a urea cycle disorder. Methods and compositions are also provided for modulating genes encoding enzymes that participate in the urea cycle by altering gene signaling networks.
Claims27
A method for increasing OTC gene expression in a cell harboring an OTC mutation associated with a partial reduction of OTC function, comprising:
- contacting the cell with an effective amount of a compound that inhibits a target selected from the group consisting of JAK1, JAK2, JAK3, HSP90, MAPK, EGFR, FGFR, BRAF, RAF1, KDR, FLT1, TBK1, IKBKE, PRKAA1, PRKAA2, PRKAB1, BMPR1A and BMPR1B .
A method for increasing OTC gene expression in a cell harboring an OTC mutation associated with a partial reduction of OTC function, comprising:
- contacting the cell with an siRNA compound that inhibits a target selected from the group consisting of JAK1, WSTR1, YAP1, CSF1R, LYN, SMAD3, NTRK1, EPHB3, EPHB4, FGFR4, INSR, KDR, FLT1, FGFR2, EPHB2, PDGFRB, IRF5, FGFR1, EPHB1, FYN, FLT4, YY1, IRF1, IGF-1, SMAD1, DDR1, HSP90AA1, and SMAD2.
A method for increasing OTC expression in a human subject harboring an OTC mutation associated with a partial reduction of OTC function, comprising:
- administering to the subject an effective amount of a compound that inhibits a target selected from the group consisting of JAK1, JAK2, JAK3, HSP90, MAPK, EGFR, FGFR, BRAF, RAF1, KDR, FLT1, TBK1, IKBKE, PRKAA1, PRKAA2, PRKAB1, BMPR1A and BMPR1B.
Dependencies are inferred from the claim text.