| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a5cd346a1a1d09725e016bdbde6dba04 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4702
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-08
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-67
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-705
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-67
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-63
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
| filingDate |
2022-04-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_00b8120d78cfe93281a99baa9384d9b0
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d6359c9dcf2ff36daafc2929128d3200 |
| publicationDate |
2022-10-13-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2022214635-A1 |
| titleOfInvention |
Nucleic acid molecules for compensation of stxbp1 haploinsufficiency and their use in the treatment of stxbp1-related disorders |
| abstract |
The present invention relates to nucleic acid molecules comprising a target binding sequence complementary to a part of the STXBP1 mRNA sequence and an effector domain a SINE B2, which nucleic acid molecule can upregulate protein translation of the target STXBP1 mRNA. The invention further relates to expression constructs, such gene therapy vectors, for expression of the nucleic acid molecules of the invention, and to composition comprising the nucleic acid molecules and expression constructs of the invention. The invention further discloses methods wherein the nucleic acid molecules, expression constructs and compositions of the invention are used for compensation of STXBP1 haploinsufficiency in the prevention or treatment of STXBP1-related disorders. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2023073071-A1 |
| priorityDate |
2021-04-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |