Gene therapy vectors for treatment of Danon Disease
The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B,...
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 | KERAVALA, Annahita PRATUMSUWAN, Piratip SHAH, Gaurav WONG, Roderick PRABHAKAR, Raj YALAMANCHI, Naveen |
| description | The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease. |
| format | Patent |
| fullrecord | <record><control><sourceid>epo_EVB</sourceid><recordid>TN_cdi_epo_espacenet_AU2023201237A1</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>AU2023201237A1</sourcerecordid><originalsourceid>FETCH-epo_espacenet_AU2023201237A13</originalsourceid><addsrcrecordid>eNrjZDB2T81LVSjJSC1KLKhUKEtNLskvKlZIyy9SKClKTSzJTc0rUchPU3BJzMvPU3DJLE5NLE7lYWBNS8wpTuWF0twMym6uIc4euqkF-fGpxQWJyUAzS-IdQ40MjIyNDAyNjM0dDY2JUwUAiGMs1g</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>patent</recordtype></control><display><type>patent</type><title>Gene therapy vectors for treatment of Danon Disease</title><source>esp@cenet</source><creator>KERAVALA, Annahita ; PRATUMSUWAN, Piratip ; SHAH, Gaurav ; WONG, Roderick ; PRABHAKAR, Raj ; YALAMANCHI, Naveen</creator><creatorcontrib>KERAVALA, Annahita ; PRATUMSUWAN, Piratip ; SHAH, Gaurav ; WONG, Roderick ; PRABHAKAR, Raj ; YALAMANCHI, Naveen</creatorcontrib><description>The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.</description><language>eng</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; ENZYMOLOGY ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; ORGANIC CHEMISTRY ; PEPTIDES ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPIRITS ; VINEGAR ; WINE</subject><creationdate>2023</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=20230413&DB=EPODOC&CC=AU&NR=2023201237A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25564,76547</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20230413&DB=EPODOC&CC=AU&NR=2023201237A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>KERAVALA, Annahita</creatorcontrib><creatorcontrib>PRATUMSUWAN, Piratip</creatorcontrib><creatorcontrib>SHAH, Gaurav</creatorcontrib><creatorcontrib>WONG, Roderick</creatorcontrib><creatorcontrib>PRABHAKAR, Raj</creatorcontrib><creatorcontrib>YALAMANCHI, Naveen</creatorcontrib><title>Gene therapy vectors for treatment of Danon Disease</title><description>The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.</description><subject>BEER</subject><subject>BIOCHEMISTRY</subject><subject>CHEMISTRY</subject><subject>COMPOSITIONS THEREOF</subject><subject>CULTURE MEDIA</subject><subject>ENZYMOLOGY</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>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</subject><subject>SPIRITS</subject><subject>VINEGAR</subject><subject>WINE</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2023</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZDB2T81LVSjJSC1KLKhUKEtNLskvKlZIyy9SKClKTSzJTc0rUchPU3BJzMvPU3DJLE5NLE7lYWBNS8wpTuWF0twMym6uIc4euqkF-fGpxQWJyUAzS-IdQ40MjIyNDAyNjM0dDY2JUwUAiGMs1g</recordid><startdate>20230413</startdate><enddate>20230413</enddate><creator>KERAVALA, Annahita</creator><creator>PRATUMSUWAN, Piratip</creator><creator>SHAH, Gaurav</creator><creator>WONG, Roderick</creator><creator>PRABHAKAR, Raj</creator><creator>YALAMANCHI, Naveen</creator><scope>EVB</scope></search><sort><creationdate>20230413</creationdate><title>Gene therapy vectors for treatment of Danon Disease</title><author>KERAVALA, Annahita ; PRATUMSUWAN, Piratip ; SHAH, Gaurav ; WONG, Roderick ; PRABHAKAR, Raj ; YALAMANCHI, Naveen</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_AU2023201237A13</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng</language><creationdate>2023</creationdate><topic>BEER</topic><topic>BIOCHEMISTRY</topic><topic>CHEMISTRY</topic><topic>COMPOSITIONS THEREOF</topic><topic>CULTURE MEDIA</topic><topic>ENZYMOLOGY</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>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>SPIRITS</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>KERAVALA, Annahita</creatorcontrib><creatorcontrib>PRATUMSUWAN, Piratip</creatorcontrib><creatorcontrib>SHAH, Gaurav</creatorcontrib><creatorcontrib>WONG, Roderick</creatorcontrib><creatorcontrib>PRABHAKAR, Raj</creatorcontrib><creatorcontrib>YALAMANCHI, Naveen</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>KERAVALA, Annahita</au><au>PRATUMSUWAN, Piratip</au><au>SHAH, Gaurav</au><au>WONG, Roderick</au><au>PRABHAKAR, Raj</au><au>YALAMANCHI, Naveen</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Gene therapy vectors for treatment of Danon Disease</title><date>2023-04-13</date><risdate>2023</risdate><abstract>The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.</abstract><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext_linktorsrc |
| identifier | |
| ispartof | |
| issn | |
| language | eng |
| recordid | cdi_epo_espacenet_AU2023201237A1 |
| source | esp@cenet |
| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE |
| title | Gene therapy vectors for treatment of Danon Disease |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-21T17%3A16%3A04IST&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=KERAVALA,%20Annahita&rft.date=2023-04-13&rft_id=info:doi/&rft_dat=%3Cepo_EVB%3EAU2023201237A1%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 |