COMPOSITIONS, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9, LDLR, AND/OR ANGPTL3
Provided herein are compositions, systems, and methods for modulating gene expression. Also provided herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat famil...
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| creator | DE L'HORTET, Alexandra Sylvie Collin LIU, Yanxia KO, Tengyu JADHAV, Kavita BOREGOWDA, Siddaraju Valagerehalli PHAM, Annie SALZMAN, Amber Ruth YANG, Guang |
| description | Provided herein are compositions, systems, and methods for modulating gene expression. Also provided herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat familial hypercholesterolemia. In some embodiments, the compositions, systems, and methods provided herein involve suppression of endogenous PCSK9 and/or activation of endogenous LDLR. In some embodiments, the compositions, systems, and methods provided herein involve two or more of: suppression of endogenous PCSK9, activation of endogenous LDLR, and/or suppression of ANGPTL3.
La présente invention concerne des compositions, des systèmes et des procédés permettant de moduler l'expression génique. L'invention concerne également des systèmes et des méthodes de traitement d'une maladie ou d'une affection par modulation de l'expression génique. Dans certains modes de réalisation, les compositions, les systèmes et les procédés et les méthodes de l'invention sont utilisés pour traiter l'hypercholestérolémie familiale. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention impliquent la suppression de PCSK9 endogène et/ou l'activation de LDLR endogène. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de la présente invention impliquent au moins deux parmi : la suppression de PCSK9 endogène, l'activation de LDLR endogène, et/ou la suppression de ANGPTL3. |
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La présente invention concerne des compositions, des systèmes et des procédés permettant de moduler l'expression génique. L'invention concerne également des systèmes et des méthodes de traitement d'une maladie ou d'une affection par modulation de l'expression génique. Dans certains modes de réalisation, les compositions, les systèmes et les procédés et les méthodes de l'invention sont utilisés pour traiter l'hypercholestérolémie familiale. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention impliquent la suppression de PCSK9 endogène et/ou l'activation de LDLR endogène. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de la présente invention impliquent au moins deux parmi : la suppression de PCSK9 endogène, l'activation de LDLR endogène, et/ou la suppression de ANGPTL3.</description><language>eng ; fre</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 ; 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>2024</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=20240919&DB=EPODOC&CC=WO&NR=2024191731A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,777,882,25545,76296</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20240919&DB=EPODOC&CC=WO&NR=2024191731A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>DE L'HORTET, Alexandra Sylvie Collin</creatorcontrib><creatorcontrib>LIU, Yanxia</creatorcontrib><creatorcontrib>KO, Tengyu</creatorcontrib><creatorcontrib>JADHAV, Kavita</creatorcontrib><creatorcontrib>BOREGOWDA, Siddaraju Valagerehalli</creatorcontrib><creatorcontrib>PHAM, Annie</creatorcontrib><creatorcontrib>SALZMAN, Amber Ruth</creatorcontrib><creatorcontrib>YANG, Guang</creatorcontrib><title>COMPOSITIONS, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9, LDLR, AND/OR ANGPTL3</title><description>Provided herein are compositions, systems, and methods for modulating gene expression. Also provided herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat familial hypercholesterolemia. In some embodiments, the compositions, systems, and methods provided herein involve suppression of endogenous PCSK9 and/or activation of endogenous LDLR. In some embodiments, the compositions, systems, and methods provided herein involve two or more of: suppression of endogenous PCSK9, activation of endogenous LDLR, and/or suppression of ANGPTL3.
La présente invention concerne des compositions, des systèmes et des procédés permettant de moduler l'expression génique. L'invention concerne également des systèmes et des méthodes de traitement d'une maladie ou d'une affection par modulation de l'expression génique. Dans certains modes de réalisation, les compositions, les systèmes et les procédés et les méthodes de l'invention sont utilisés pour traiter l'hypercholestérolémie familiale. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention impliquent la suppression de PCSK9 endogène et/ou l'activation de LDLR endogène. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de la présente invention impliquent au moins deux parmi : la suppression de PCSK9 endogène, l'activation de LDLR endogène, et/ou la suppression de ANGPTL3.</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>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>2024</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNqNjL0KwjAURrs4iPoOF1wrmlaQjtc0bYL5IwlIp1IkTqKFis9vKD6A0_mG851l9qFGWeNFEEb7HHznA1NpoK5BscBN7aExDoJjGIRuoUElpEAJvLPMUW4kSxeXoATCuYOArmWzaqm_VDnIWro5uE8d1K0Nslxni_vwmOLmx1W2bVigfBfHVx-ncbjFZ3z3V1MciiOpyKkkSMr_rC8ZnTo_</recordid><startdate>20240919</startdate><enddate>20240919</enddate><creator>DE L'HORTET, Alexandra Sylvie Collin</creator><creator>LIU, Yanxia</creator><creator>KO, Tengyu</creator><creator>JADHAV, Kavita</creator><creator>BOREGOWDA, Siddaraju Valagerehalli</creator><creator>PHAM, Annie</creator><creator>SALZMAN, Amber Ruth</creator><creator>YANG, Guang</creator><scope>EVB</scope></search><sort><creationdate>20240919</creationdate><title>COMPOSITIONS, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9, LDLR, AND/OR ANGPTL3</title><author>DE L'HORTET, Alexandra Sylvie Collin ; LIU, Yanxia ; KO, Tengyu ; JADHAV, Kavita ; BOREGOWDA, Siddaraju Valagerehalli ; PHAM, Annie ; SALZMAN, Amber Ruth ; YANG, Guang</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_WO2024191731A13</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; fre</language><creationdate>2024</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>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>DE L'HORTET, Alexandra Sylvie Collin</creatorcontrib><creatorcontrib>LIU, Yanxia</creatorcontrib><creatorcontrib>KO, Tengyu</creatorcontrib><creatorcontrib>JADHAV, Kavita</creatorcontrib><creatorcontrib>BOREGOWDA, Siddaraju Valagerehalli</creatorcontrib><creatorcontrib>PHAM, Annie</creatorcontrib><creatorcontrib>SALZMAN, Amber Ruth</creatorcontrib><creatorcontrib>YANG, Guang</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>DE L'HORTET, Alexandra Sylvie Collin</au><au>LIU, Yanxia</au><au>KO, Tengyu</au><au>JADHAV, Kavita</au><au>BOREGOWDA, Siddaraju Valagerehalli</au><au>PHAM, Annie</au><au>SALZMAN, Amber Ruth</au><au>YANG, Guang</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>COMPOSITIONS, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9, LDLR, AND/OR ANGPTL3</title><date>2024-09-19</date><risdate>2024</risdate><abstract>Provided herein are compositions, systems, and methods for modulating gene expression. Also provided herein are systems and methods for treating a disease or a condition by modulating gene expression. In some embodiments, the compositions, systems, and methods provided herein are used to treat familial hypercholesterolemia. In some embodiments, the compositions, systems, and methods provided herein involve suppression of endogenous PCSK9 and/or activation of endogenous LDLR. In some embodiments, the compositions, systems, and methods provided herein involve two or more of: suppression of endogenous PCSK9, activation of endogenous LDLR, and/or suppression of ANGPTL3.
La présente invention concerne des compositions, des systèmes et des procédés permettant de moduler l'expression génique. L'invention concerne également des systèmes et des méthodes de traitement d'une maladie ou d'une affection par modulation de l'expression génique. Dans certains modes de réalisation, les compositions, les systèmes et les procédés et les méthodes de l'invention sont utilisés pour traiter l'hypercholestérolémie familiale. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de l'invention impliquent la suppression de PCSK9 endogène et/ou l'activation de LDLR endogène. Selon certains modes de réalisation, les compositions, les systèmes et les procédés de la présente invention impliquent au moins deux parmi : la suppression de PCSK9 endogène, l'activation de LDLR endogène, et/ou la suppression de ANGPTL3.</abstract><oa>free_for_read</oa></addata></record> |
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| 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 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, SYSTEMS, AND METHODS FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA BY TARGETING PCSK9, LDLR, AND/OR ANGPTL3 |
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