http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3570895-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_e3b4f2ef8f062dcde3a2b7cd81748472 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_43a72884e25aaecffebe0942132d704f http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_37f4922dfb7777b019e504b885211b8e |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-0058 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07K14-72 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 |
| filingDate | 2018-01-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_84300d49f7a7099b18bd2e9fe3acde62 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_841d9c9a9dde2d2d49b7f3d70a6861e7 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d858e28a70c894bd1fb6bef57795e45d http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_23889bf7046eb54003c36bda8274c7c2 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4e3ec835495313509dcb78099ec93c42 |
| publicationDate | 2019-11-27-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | EP-3570895-A1 |
| titleOfInvention | Methods of expressing a polynucleotide of interest in the cone photoreceptors |
| abstract | Intraocular injection of adeno-associated viral (AAV) vectors has been an evident route for delivering gene drugs into the retina. Currently, the vectors need to be injected into the subretinal space in order to provide gene delivery to cones. In this approach, gene delivery is limited to cells that contact the local "bleb" of injected fluid. Furthermore, retinal detachment that occurs during subretinal injections is a concern in eyes with retinal degeneration. Here, the inventors establish several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea with supporting studies in mouse models, human induced pluripotent stem cell-derived organoids, post-mortem human retinal explants and living macaques. They show that an engineered AAV2 variant provides gene delivery to foveal cones with a well-tolerated dose administered intravitreally. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Accordingly, the present invention relates to method of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising intravitreal delivery of a therapeutically effective amount of a recombinant AAV2-derived vector comprising a VP1 capsid protein as set forth in SEQ ID NO: 1 and the polynucleotide of interest under the control of the PR1.7 promoter as set forth in SEQ ID NO:2. |
| priorityDate | 2017-01-17-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
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