http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2016068838-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_1763f7f78d01cad638210d060676591a |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3233 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
| filingDate | 2014-04-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0db62add85439e4d8d4bc3f793cc655d http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_477df33e72be9a34ec03d300f9159a8f |
| publicationDate | 2016-03-10-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | US-2016068838-A1 |
| titleOfInvention | SMN2 Element 1 Antisense Compositions and Methods and Uses Thereof |
| abstract | The invention provides methods and compositions for treatment of spinal muscular atrophy (SMA). In one aspect of the invention, a series of compositions comprising an antisense oligonucleotide targeting the Element 1 site on the SMN2 pre-mRNA and a Morpholino backbone is disclosed. In another aspect of the invention, a method of treating SMA patients by modulating the splicing of SMN2 pre-mRNA to increase the amount of full-length SMN is disclosed. Certain embodiments of the inventive method comprise administering an E1-targeting antisense oligonucleotide, such as Morpholino based antisense oligonucleotide, to a SMA subject. |
| isCitedBy | http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11299737-B1 |
| priorityDate | 2013-04-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 77.