OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disc...
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| creator | Butler, David Charles Donnell Lu, Genliang Iwamoto, Naoki Zhang, Jason Jingxin Wisniewska-wrona, Hanna Maria Frank-Kamenetsky, Maria David Cheng, Xiayun Vargeese, Chandra Cho, Young Jin Marappan, Subramanian Apponi, Luciano Henrique Vathipadiekal, Vinod |
| description | Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown. |
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In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.</description><language>eng</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; DERIVATIVES THEREOF ; ENZYMOLOGY ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; NUCLEIC ACIDS ; NUCLEOSIDES ; NUCLEOTIDES ; ORGANIC CHEMISTRY ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPIRITS ; SUGARS ; 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=20240404&DB=EPODOC&CC=US&NR=2024109931A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25563,76318</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20240404&DB=EPODOC&CC=US&NR=2024109931A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>Butler, David Charles Donnell</creatorcontrib><creatorcontrib>Lu, Genliang</creatorcontrib><creatorcontrib>Iwamoto, Naoki</creatorcontrib><creatorcontrib>Zhang, Jason Jingxin</creatorcontrib><creatorcontrib>Wisniewska-wrona, Hanna Maria</creatorcontrib><creatorcontrib>Frank-Kamenetsky, Maria David</creatorcontrib><creatorcontrib>Cheng, Xiayun</creatorcontrib><creatorcontrib>Vargeese, Chandra</creatorcontrib><creatorcontrib>Cho, Young Jin</creatorcontrib><creatorcontrib>Marappan, Subramanian</creatorcontrib><creatorcontrib>Apponi, Luciano Henrique</creatorcontrib><creatorcontrib>Vathipadiekal, Vinod</creatorcontrib><title>OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF</title><description>Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. 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In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA DERIVATIVES THEREOF ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NUCLEIC ACIDS NUCLEOSIDES NUCLEOTIDES ORGANIC CHEMISTRY PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS SUGARS VINEGAR WINE |
| title | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF |
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