Compositions and methods for treating beta-hemoglobinopathies
The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In s...
Gespeichert in:
| Hauptverfasser: | , , , , , |
|---|---|
| Format: | Patent |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext bestellen |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | |
|---|---|
| container_issue | |
| container_start_page | |
| container_title | |
| container_volume | |
| creator | Ahlers, Jeffrey Bartlett, Jeffrey Symonds, Geoffrey Phillip Lee, Chi-Lin Ringpis, Gene-Errol Eugenio Yan, Ming |
| description | The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof. |
| format | Patent |
| fullrecord | <record><control><sourceid>epo_EVB</sourceid><recordid>TN_cdi_epo_espacenet_US10961537B2</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>US10961537B2</sourcerecordid><originalsourceid>FETCH-epo_espacenet_US10961537B23</originalsourceid><addsrcrecordid>eNrjZLB1zs8tyC_OLMnMzytWSMxLUchNLcnITylWSMsvUigpSk0sycxLV0hKLUnUzUjNzU_PyU_KzMsvSCzJyEwt5mFgTUvMKU7lhdLcDIpuriHOHrqpBfnxqcUFicmpeakl8aHBhgaWZoamxuZORsbEqAEAdTgxFQ</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>patent</recordtype></control><display><type>patent</type><title>Compositions and methods for treating beta-hemoglobinopathies</title><source>esp@cenet</source><creator>Ahlers, Jeffrey ; Bartlett, Jeffrey ; Symonds, Geoffrey Phillip ; Lee, Chi-Lin ; Ringpis, Gene-Errol Eugenio ; Yan, Ming</creator><creatorcontrib>Ahlers, Jeffrey ; Bartlett, Jeffrey ; Symonds, Geoffrey Phillip ; Lee, Chi-Lin ; Ringpis, Gene-Errol Eugenio ; Yan, Ming</creatorcontrib><description>The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.</description><language>eng</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; ENZYMOLOGY ; HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; ORGANIC CHEMISTRY ; PEPTIDES ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS ; SPIRITS ; VINEGAR ; WINE</subject><creationdate>2021</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20210330&DB=EPODOC&CC=US&NR=10961537B2$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25564,76547</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20210330&DB=EPODOC&CC=US&NR=10961537B2$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>Ahlers, Jeffrey</creatorcontrib><creatorcontrib>Bartlett, Jeffrey</creatorcontrib><creatorcontrib>Symonds, Geoffrey Phillip</creatorcontrib><creatorcontrib>Lee, Chi-Lin</creatorcontrib><creatorcontrib>Ringpis, Gene-Errol Eugenio</creatorcontrib><creatorcontrib>Yan, Ming</creatorcontrib><title>Compositions and methods for treating beta-hemoglobinopathies</title><description>The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.</description><subject>BEER</subject><subject>BIOCHEMISTRY</subject><subject>CHEMISTRY</subject><subject>COMPOSITIONS THEREOF</subject><subject>CULTURE MEDIA</subject><subject>ENZYMOLOGY</subject><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>MICROBIOLOGY</subject><subject>MICROORGANISMS OR ENZYMES</subject><subject>MUTATION OR GENETIC ENGINEERING</subject><subject>ORGANIC CHEMISTRY</subject><subject>PEPTIDES</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><subject>SPIRITS</subject><subject>VINEGAR</subject><subject>WINE</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2021</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZLB1zs8tyC_OLMnMzytWSMxLUchNLcnITylWSMsvUigpSk0sycxLV0hKLUnUzUjNzU_PyU_KzMsvSCzJyEwt5mFgTUvMKU7lhdLcDIpuriHOHrqpBfnxqcUFicmpeakl8aHBhgaWZoamxuZORsbEqAEAdTgxFQ</recordid><startdate>20210330</startdate><enddate>20210330</enddate><creator>Ahlers, Jeffrey</creator><creator>Bartlett, Jeffrey</creator><creator>Symonds, Geoffrey Phillip</creator><creator>Lee, Chi-Lin</creator><creator>Ringpis, Gene-Errol Eugenio</creator><creator>Yan, Ming</creator><scope>EVB</scope></search><sort><creationdate>20210330</creationdate><title>Compositions and methods for treating beta-hemoglobinopathies</title><author>Ahlers, Jeffrey ; Bartlett, Jeffrey ; Symonds, Geoffrey Phillip ; Lee, Chi-Lin ; Ringpis, Gene-Errol Eugenio ; Yan, Ming</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_US10961537B23</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng</language><creationdate>2021</creationdate><topic>BEER</topic><topic>BIOCHEMISTRY</topic><topic>CHEMISTRY</topic><topic>COMPOSITIONS THEREOF</topic><topic>CULTURE MEDIA</topic><topic>ENZYMOLOGY</topic><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>MICROBIOLOGY</topic><topic>MICROORGANISMS OR ENZYMES</topic><topic>MUTATION OR GENETIC ENGINEERING</topic><topic>ORGANIC CHEMISTRY</topic><topic>PEPTIDES</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><topic>SPIRITS</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>Ahlers, Jeffrey</creatorcontrib><creatorcontrib>Bartlett, Jeffrey</creatorcontrib><creatorcontrib>Symonds, Geoffrey Phillip</creatorcontrib><creatorcontrib>Lee, Chi-Lin</creatorcontrib><creatorcontrib>Ringpis, Gene-Errol Eugenio</creatorcontrib><creatorcontrib>Yan, Ming</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>Ahlers, Jeffrey</au><au>Bartlett, Jeffrey</au><au>Symonds, Geoffrey Phillip</au><au>Lee, Chi-Lin</au><au>Ringpis, Gene-Errol Eugenio</au><au>Yan, Ming</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Compositions and methods for treating beta-hemoglobinopathies</title><date>2021-03-30</date><risdate>2021</risdate><abstract>The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.</abstract><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext_linktorsrc |
| identifier | |
| ispartof | |
| issn | |
| language | eng |
| recordid | cdi_epo_espacenet_US10961537B2 |
| source | esp@cenet |
| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE |
| title | Compositions and methods for treating beta-hemoglobinopathies |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-28T15%3A58%3A58IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-epo_EVB&rft_val_fmt=info:ofi/fmt:kev:mtx:patent&rft.genre=patent&rft.au=Ahlers,%20Jeffrey&rft.date=2021-03-30&rft_id=info:doi/&rft_dat=%3Cepo_EVB%3EUS10961537B2%3C/epo_EVB%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_id=info:pmid/&rfr_iscdi=true |