Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_d6559372bde7c7c71d22286457328c86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_9d6f7da1e425c8a3ef1da21e08f4c196 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-52 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-48 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-40 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2830-50 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2740-15043 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-867 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P7-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-867 |
filingDate |
2016-01-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c6e0ba6dc25ccfb86740464e3f363ca7 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_3b9af09dd9a29bbe8e242d4986d2e032 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_402616a69741630a1ea7a8aaa107a314 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_a9fc840990ff5eae8ec0475062901153 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_8c6377eafc87fbdff514a248a282c515 |
publicationDate |
2017-09-07-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
AU-2016209313-A1 |
titleOfInvention |
Viral vectors for prophylaxis and therapy of hemoglobinopathies |
abstract |
Provided are compositions and methods for inducing expression of human beta-globin in erythrocytes for use in prophylaxis and/or therapy of a hemoglobinopathy in an individual. The method generally entails introducing into CD34+ cells a polynucleotide encoding: i) a 5' long terminal repeat (LTR) and a self-inactivating 3' LTR; ii) at least one polyadenylation signal; iii) at least one promoter; iv) a globin gene locus control region (LCR); v) an ankyrin insulator element (Ank); vi) a Woodchuck Post-Regulatory Element (WPRE) configured such that the WPRE does not integrate into a target genome; and vii) a sequence that is a reverse complement of a sequence encoding human beta-globin, and can include beta-globin that has a PT87Q mutation. Intron 2 of the beta globin gene can be a complete intron. Modified erythrocyte progenitor cells, recombinant vectors and virions comprising recombinant polynucleotides, and methods of making the vectors and virions are included. |
priorityDate |
2015-01-21-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |