| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_914ce1e64249e3dc0af1b375f3920f5d
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2de66e82adda02ef6bb18a5fd58febd7 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2217-072
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2227-105
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4702
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4703
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-14
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0066
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-001
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0085
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-47
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
| filingDate |
2018-03-23-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_11de42de55296b6398c94a3625afd104
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f0a4a73dfa4d42b97b3736327dd075d0
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c178ffbdfde8ff2f67635bf190fcfd9b
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d9a58265849a6b85b29c59fb3c484758 |
| publicationDate |
2018-09-27-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2018172795-A1 |
| titleOfInvention |
MeCP2 EXPRESSION CASSETTES |
| abstract |
MECP2 EXPRESSION CASSETTES. The present invention provides nucleic acid molecules comprising a MeCP2 expression cassette, the expression cassette comprising, in operable linkage from 5' to 3': a 5' transcriptional control region comprising a promoter capable of driving transcription in neural cells; an open reading frame encoding a MeCP2 protein; translation control signals; a 3' untranslated region (3'UTR) comprising one or more of: (i) a binding site for mir-22; (ii) a binding site for mir-19; (iii) a binding site for miR-132; (iv) a binding site for miR124; and (v) an AU-rich element; and transcriptional termination signals; wherein the MeCP2 expression cassette is not more than about 5 kb in length. The invention further provides viral vectors, especially vectors derived from adeno-associated virus (AAV), for use in therapeutic delivery of such expression cassettes. The nucleic acid molecules and viral vectors disclosed herein provide novel tools for expressing MeCP2 and are of particular value in the treatment of disorders associated with reduced MeCP2 activity, including Rett syndrome. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022094078-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021113634-A1
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-114026236-A
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020047234-A1 |
| priorityDate |
2017-03-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |