http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2018215736-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_c9afda3a68e2c7e0e631e733efa5b735 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D403-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D403-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D401-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D401-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07D403-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07D403-12 |
| filingDate | 2015-08-10-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_90069c97a3d94b728e9847d7058fbce9 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_77cfcafded43610e89597a7fe5144d6b http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4058c32b2fccc870f109c710b009db80 |
| publicationDate | 2018-08-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | US-2018215736-A1 |
| titleOfInvention | Bisamidinium-based inhibitors for the treatment of myotonic dystrophy |
| abstract | The invention provides compounds, compositions and therapeutic methods. The compounds and compositions can be used for the treatment of myotonic dystrophy. The compounds can selectively bind to CUG repeats in RNA, or to CTG repeats in DNA, and inhibit replication of the nucleic acids. RNA-targeted therapeutic agents for the treatment of myotonic dystrophy type 1 (DM1) are described. In one embodiment, two bisamidinium ligands are linked using “click” chemistry to form a heterodimer that is a potent inhibitor of the MBNL1-rCUG exp complex (K I =25±8 nM), is relatively non-toxic to HeLa cells, dissolves nuclear foci, corrects >80% of the IR misregulated alternative splicing in DM1 model cells (1 μM), and shows improvement of disease phenotypes in a DM1 Drosophila model. |
| priorityDate | 2014-08-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 132.