http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2018035311-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_d5647c2be83c84110a7587b5319d4472 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-67 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07H21-02 |
| filingDate | 2017-08-17-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3a5725dc7cfa1008de5316a29ff42762 |
| publicationDate | 2018-02-22-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | WO-2018035311-A1 |
| titleOfInvention | Compositions and methods for modulating gene expression using reading frame surveillance |
| abstract | The invention provides compositions and methods for stimulating or suppressing the expression of a gene of interest, as well for inducing alternative RNA splicing of a target gene. The compositions and methods described herein can be used, for instance, to produce genes and RNA equivalents optimized for expression in a particular cell type. The invention additionally features nucleic acid duplexes capable of promoting gene expression upon delivery into a cell, such as a mammalian cell described herein. The compositions and methods described herein can additionally be used to silence gene expression, as well as to stimulate alternative splicing of a pre-mRNA transcript, for instance, by way of exon skipping. The invention additionally provides methods of treating a variety of genetic disorders. Exemplary diseases that can be treated using genes optimized using the compositions and methods described herein include recessive genetic disorders, such as X-linked myotubular myopathy, Pompe disease, recessive catecholaminergic polymorphic ventricular tachycardia, and Crigler-Najjar syndrome, among others, as well as disorders associated with deleterious frameshift mutations, such as Duchenne muscular dystrophy. |
| isCitedBy | http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021025725-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022062440-A1 |
| priorityDate | 2016-08-19-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 1554.