http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021127655-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a6748c1fba1292abba86450fa886727c |
| classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-107 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2840-203 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2217-075 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2227-105 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A01K2267-0306 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P21-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-47 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07K14-4707 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-85 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-86 |
| filingDate | 2020-12-21^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_223aa954a480d701fc15c05b7e9cbbd1 |
| publicationDate | 2021-06-24^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | WO-2021127655-A1 |
| titleOfInvention | Optimized gene therapy for targeting muscle in muscle diseases |
| abstract | The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), optimized for delivering a transgene to muscles. The optimized vectors contain constitutive or a muscle-specific promoter to deliver whole body or skeletal/heart muscle-specific transgene expression, respectively, in combination with a transgene cDNA to replace the gene mutation found in a muscle disease with a normal copy of the gene, an internal ribosomal entry site (IRES) to allow for production of a second protein from the same transcript, and a muscle growth factor, to build new muscle growth and strength. For example, the invention provides The disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV), designed for treatment of GNE myopathy in which the rAAV expresses UDP-GlcNAc-epimerase/ManNAc-6 alone or in combination with a muscle growth factor or muscle transdifferentation factor. The provided AAV replace the mutated GNE gene expression while expressing proteins that stimulate muscle growth. |
| isCitedBy | http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2023076972-A1 |
| priorityDate | 2019-12-20^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
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