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US 2023/0054976 A1Published application
METHODS FOR INHIBITION OF ALPHA-SYNUCLEIN mRNA USING SMALL MOLECULES
AssigneesThe Scripps Research Institute, Rutgers, The State University of New Jersey
InventorsMatthew David DISNEY, M. Maral MOURADIAN
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Abstract
A sequence-based design has provided a group of small molecules that target the IRE structure and inhibit SNCA translation in cells, which are named synucleozid compounds. The synucleozid compounds have a diphenyloxo- or diphenylamino-benzimidazole or indole core with various terminal amino or heterocycle groups as substituents.
Claims33
A method for complexing an SNCA mRNA comprising contacting the SNCA mRNA with a synucleozid compound comprising Formula I
- wherein:
- Y is nitrogen or CR1
- X is oxygen or NR2;
- Z is hydrogen, methyl or
- Im1 is guanidyl
- imidazolyl, dihydroimidazolyl, imidazolinyl, pyrrolyl, pyrrolidinyl, or cyano;
- Im2 is guanidyl, imidazolyl, dihydroimidazolyl, imidazolinyl, pyrrolyl, pyrrolidinyl, or cyano;
- Im1 and Im2 are the same or different;
- R1 and R2 are each independently hydrogen or methyl;
- and a pharmaceutically acceptable salt thereof.
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Dependencies are inferred from the claim text.