Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_f9bc9018b72f6eb59442a1fb91dd5e2c |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14171 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14121 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y310-01001 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-46 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K47-26 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-14 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-761 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K35-76 |
filingDate |
2013-06-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_dba95a25ad42b0c386d0d7b4d97a0972 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_84797b89b270d4db3632eeb4d9426c9e |
publicationDate |
2013-12-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
US-2013323207-A1 |
titleOfInvention |
Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
abstract |
The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering α-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2016120960-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2018036388-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11253612-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-9827295-B2 |
priorityDate |
2011-06-08-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |