Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_f8c0982b0f02b1ed6d20a315370a3a3a http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_f40cd55e614d5fad02d84391df2c885d http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_fd522c113ab9706761479770dadf1a82 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_3c9c7aedb976d7e01f6addb5566c7436 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/G01N2500-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/G01N2500-04 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D403-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D495-04 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D519-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/G01N33-5308 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-10 |
filingDate |
2014-07-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_17d8f6b6f8a6489275112249090eba93 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_dade1fadae9bf9f14c9e73680fb55b4d http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_da6b5c55d2f9acc05718ad61d2622395 |
publicationDate |
2015-01-22-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2015009678-A2 |
titleOfInvention |
Specific targeting of rna expanded repeat sequences |
abstract |
The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG) exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG) exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS); to methods of making the small molecule compounds; and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo , using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017049165-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022051405-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10118920-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022021981-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10428065-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021087084-A1 |
priorityDate |
2013-07-15-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |