http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-2008523093-A
Outgoing Links
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classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-42 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2840-203 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-85 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 |
classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-16 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-00 |
classificationIPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 |
classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K35-76 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-43 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-16 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-00 |
filingDate | 2005-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationDate | 2008-07-03-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber | JP-2008523093-A |
titleOfInvention | Regulated expression of transgenes in the mammalian central nervous system |
abstract | Recombinant adeno-associated virus (rAAV) vectors are provided for delivering regulatable transgenes to the mammalian central nervous system (CNS). Also provided are methods for treating patients with neurodegenerative diseases using vectors, and kits for constructing or using vectors, or for performing the methods of the invention. The transgene sequence is expressed from a promoter / enhancer region that contains one or more binding sites for transcription factors that respond to small molecule derivatives. Both the transgene construct and the construct containing the transcription factor are delivered to the target cells. Regulatable transgenes are delivered as transcription factors on the same rAAV vector or on separate vectors. A transcription factor may comprise two polypeptide chains, eg, a DNA binding domain and a transcriptional activation domain, that are active in the presence of a dimerization product such as rapamycin or a non-immunogenic analog thereof. A dimer is formed. The vectors, methods and kits of the invention may be used to deliver genes such as AADC or GDNF to the brain of patients with neurodegenerative diseases such as Parkinson's disease, where expression of AADC or GDNF in the brain is later Modulation is possible by treating the patient with rapamycin or a rapamycin analog. |
isCitedBy | http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-7212378-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-2017532966-A http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-7023108-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11578340-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/JP-2021010372-A http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11542525-B2 |
priorityDate | 2004-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 396.