COMPOSITIONS USEFUL FOR MODULATING SPLICING
The present application discloses compounds of Formula (I) that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Diseas...
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| creator | RATNI, Hasane KONETZKI, Ingo LUCAS, Brian LUZZIO, Michael |
| description | The present application discloses compounds of Formula (I) that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Disease).
La présente invention concerne des composés de formule (I) qui modulent l'épissage d'un pré-ARNm, codé par des gènes, et des méthodes de traitement de maladies et d'affections associées à l'expression génique ou à l'activité de protéines codées par des gènes, telles que l'ataxie spinocérébelleuse 3 (ASC3 ou maladie de Machado-Joseph). |
| format | Patent |
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La présente invention concerne des composés de formule (I) qui modulent l'épissage d'un pré-ARNm, codé par des gènes, et des méthodes de traitement de maladies et d'affections associées à l'expression génique ou à l'activité de protéines codées par des gènes, telles que l'ataxie spinocérébelleuse 3 (ASC3 ou maladie de Machado-Joseph).</description><language>eng ; fre</language><subject>CHEMISTRY ; HETEROCYCLIC COMPOUNDS ; HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; ORGANIC CHEMISTRY ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</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=20240725&DB=EPODOC&CC=WO&NR=2024155847A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,778,883,25547,76298</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20240725&DB=EPODOC&CC=WO&NR=2024155847A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>RATNI, Hasane</creatorcontrib><creatorcontrib>KONETZKI, Ingo</creatorcontrib><creatorcontrib>LUCAS, Brian</creatorcontrib><creatorcontrib>LUZZIO, Michael</creatorcontrib><title>COMPOSITIONS USEFUL FOR MODULATING SPLICING</title><description>The present application discloses compounds of Formula (I) that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Disease).
La présente invention concerne des composés de formule (I) qui modulent l'épissage d'un pré-ARNm, codé par des gènes, et des méthodes de traitement de maladies et d'affections associées à l'expression génique ou à l'activité de protéines codées par des gènes, telles que l'ataxie spinocérébelleuse 3 (ASC3 ou maladie de Machado-Joseph).</description><subject>CHEMISTRY</subject><subject>HETEROCYCLIC COMPOUNDS</subject><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>ORGANIC CHEMISTRY</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2024</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZNB29vcN8A_2DPH09wtWCA12dQv1UXDzD1Lw9XcJ9XEM8fRzVwgO8PF0BjJ4GFjTEnOKU3mhNDeDsptriLOHbmpBfnxqcUFicmpeakl8uL-RgZGJoamphYm5o6ExcaoANQYmPQ</recordid><startdate>20240725</startdate><enddate>20240725</enddate><creator>RATNI, Hasane</creator><creator>KONETZKI, Ingo</creator><creator>LUCAS, Brian</creator><creator>LUZZIO, Michael</creator><scope>EVB</scope></search><sort><creationdate>20240725</creationdate><title>COMPOSITIONS USEFUL FOR MODULATING SPLICING</title><author>RATNI, Hasane ; KONETZKI, Ingo ; LUCAS, Brian ; LUZZIO, Michael</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_WO2024155847A13</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; fre</language><creationdate>2024</creationdate><topic>CHEMISTRY</topic><topic>HETEROCYCLIC COMPOUNDS</topic><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>ORGANIC CHEMISTRY</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><toplevel>online_resources</toplevel><creatorcontrib>RATNI, Hasane</creatorcontrib><creatorcontrib>KONETZKI, Ingo</creatorcontrib><creatorcontrib>LUCAS, Brian</creatorcontrib><creatorcontrib>LUZZIO, Michael</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>RATNI, Hasane</au><au>KONETZKI, Ingo</au><au>LUCAS, Brian</au><au>LUZZIO, Michael</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>COMPOSITIONS USEFUL FOR MODULATING SPLICING</title><date>2024-07-25</date><risdate>2024</risdate><abstract>The present application discloses compounds of Formula (I) that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes, such as Spinocerebellar Ataxia 3 (SCA3 or Machado-Joseph Disease).
La présente invention concerne des composés de formule (I) qui modulent l'épissage d'un pré-ARNm, codé par des gènes, et des méthodes de traitement de maladies et d'affections associées à l'expression génique ou à l'activité de protéines codées par des gènes, telles que l'ataxie spinocérébelleuse 3 (ASC3 ou maladie de Machado-Joseph).</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | CHEMISTRY HETEROCYCLIC COMPOUNDS HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS |
| title | COMPOSITIONS USEFUL FOR MODULATING SPLICING |
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