Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2a819eda0adf22936a52362eeebb9fb4 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2740-15043 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2740-16043 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N7-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-28 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/C12N15-63 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-85 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-867 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-63 |
filingDate |
2017-12-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_806ea866e6d152e621a87b2c9f7b466d http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_fd5572d321a92ffa2c0fe3f421d86b9c http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d0d22630585b8d8775388459a9c39ad3 |
publicationDate |
2018-06-14-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2018106724-A1 |
titleOfInvention |
Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies |
abstract |
In certain embodiments an optimized derivative of the CCLc-βAS3-FB lentiviral vector termed (CCLc-mGata/ANK-CoreLCR-βAS3-FB), is provided which is capable of driving lineage-restricted expression of a beta-globin gene ( e.g. , an anti-sickling β-globin like gene (βΑ83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(~420 bp), HS3(~340 bp), HS4(~410 bp)) which can be used to replace the putative LCR HS sequences present within the "mini-LCR" (~3.6 kb reduced to ~1.2 kb) to produce an "optimized mini-LCR". |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-110564770-A http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10961537-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3720509-A4 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3931338-A4 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-110564770-B http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2020168004-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022229223-A1 |
priorityDate |
2016-12-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |