Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof
The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supp...
Gespeichert in:
| Hauptverfasser: | , , , , , , , |
|---|---|
| Format: | Patent |
| Sprache: | chi ; 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 | BOOTH, KEVIN T IVANCHENKO, MARYNA V HRVATIN, SINISA GREENBERG, MICHAEL E PETERS, COLE W. D NAGY, MARK AUREL COREY, DAVID P GRIFFITH, ERIC C |
| description | The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5' UTR, and/or GJB2 3' UTR), and a nucleotide sequence encoding a GJB2 protein. |
| format | Patent |
| fullrecord | <record><control><sourceid>epo_EVB</sourceid><recordid>TN_cdi_epo_espacenet_TW202227476A</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>TW202227476A</sourcerecordid><originalsourceid>FETCH-epo_espacenet_TW202227476A3</originalsourceid><addsrcrecordid>eNqNyrEKwjAQBuAuDqK-w7npIEgUO0dRi6MUHcs1-asBvZRc6vO7-ABO3_KNi-oKF99tEJZM7CGRWDW6wBmePiENSotk7W1JEBd9kAedL3tDLJ4GhVJ-IiF202LU8Usx-zkp5qdjfahW6GMD7dlBkJv6btbGmHJb7uzmn_MFEcYzHA</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>patent</recordtype></control><display><type>patent</type><title>Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof</title><source>esp@cenet</source><creator>BOOTH, KEVIN T ; IVANCHENKO, MARYNA V ; HRVATIN, SINISA ; GREENBERG, MICHAEL E ; PETERS, COLE W. D ; NAGY, MARK AUREL ; COREY, DAVID P ; GRIFFITH, ERIC C</creator><creatorcontrib>BOOTH, KEVIN T ; IVANCHENKO, MARYNA V ; HRVATIN, SINISA ; GREENBERG, MICHAEL E ; PETERS, COLE W. D ; NAGY, MARK AUREL ; COREY, DAVID P ; GRIFFITH, ERIC C</creatorcontrib><description>The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5' UTR, and/or GJB2 3' UTR), and a nucleotide sequence encoding a GJB2 protein.</description><language>chi ; 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>2022</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=20220716&DB=EPODOC&CC=TW&NR=202227476A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,309,781,886,25566,76549</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20220716&DB=EPODOC&CC=TW&NR=202227476A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>BOOTH, KEVIN T</creatorcontrib><creatorcontrib>IVANCHENKO, MARYNA V</creatorcontrib><creatorcontrib>HRVATIN, SINISA</creatorcontrib><creatorcontrib>GREENBERG, MICHAEL E</creatorcontrib><creatorcontrib>PETERS, COLE W. D</creatorcontrib><creatorcontrib>NAGY, MARK AUREL</creatorcontrib><creatorcontrib>COREY, DAVID P</creatorcontrib><creatorcontrib>GRIFFITH, ERIC C</creatorcontrib><title>Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof</title><description>The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5' UTR, and/or GJB2 3' UTR), and a nucleotide sequence encoding a GJB2 protein.</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>2022</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNqNyrEKwjAQBuAuDqK-w7npIEgUO0dRi6MUHcs1-asBvZRc6vO7-ABO3_KNi-oKF99tEJZM7CGRWDW6wBmePiENSotk7W1JEBd9kAedL3tDLJ4GhVJ-IiF202LU8Usx-zkp5qdjfahW6GMD7dlBkJv6btbGmHJb7uzmn_MFEcYzHA</recordid><startdate>20220716</startdate><enddate>20220716</enddate><creator>BOOTH, KEVIN T</creator><creator>IVANCHENKO, MARYNA V</creator><creator>HRVATIN, SINISA</creator><creator>GREENBERG, MICHAEL E</creator><creator>PETERS, COLE W. D</creator><creator>NAGY, MARK AUREL</creator><creator>COREY, DAVID P</creator><creator>GRIFFITH, ERIC C</creator><scope>EVB</scope></search><sort><creationdate>20220716</creationdate><title>Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof</title><author>BOOTH, KEVIN T ; IVANCHENKO, MARYNA V ; HRVATIN, SINISA ; GREENBERG, MICHAEL E ; PETERS, COLE W. D ; NAGY, MARK AUREL ; COREY, DAVID P ; GRIFFITH, ERIC C</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_TW202227476A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>chi ; eng</language><creationdate>2022</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>BOOTH, KEVIN T</creatorcontrib><creatorcontrib>IVANCHENKO, MARYNA V</creatorcontrib><creatorcontrib>HRVATIN, SINISA</creatorcontrib><creatorcontrib>GREENBERG, MICHAEL E</creatorcontrib><creatorcontrib>PETERS, COLE W. D</creatorcontrib><creatorcontrib>NAGY, MARK AUREL</creatorcontrib><creatorcontrib>COREY, DAVID P</creatorcontrib><creatorcontrib>GRIFFITH, ERIC C</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>BOOTH, KEVIN T</au><au>IVANCHENKO, MARYNA V</au><au>HRVATIN, SINISA</au><au>GREENBERG, MICHAEL E</au><au>PETERS, COLE W. D</au><au>NAGY, MARK AUREL</au><au>COREY, DAVID P</au><au>GRIFFITH, ERIC C</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof</title><date>2022-07-16</date><risdate>2022</risdate><abstract>The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5' UTR, and/or GJB2 3' UTR), and a nucleotide sequence encoding a GJB2 protein.</abstract><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext_linktorsrc |
| identifier | |
| ispartof | |
| issn | |
| language | chi ; eng |
| recordid | cdi_epo_espacenet_TW202227476A |
| 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 | Recombinant adeno associated virus (rAAV) encoding GJB2 and uses thereof |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-18T06%3A37%3A02IST&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=BOOTH,%20KEVIN%20T&rft.date=2022-07-16&rft_id=info:doi/&rft_dat=%3Cepo_EVB%3ETW202227476A%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 |