Antisense oligonucleotides targeting ACTL6B

The present invention relates to antisense oligonucleotide splicing modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP...

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Hauptverfasser:	JOENSEN LARS, VIKESA JONAS, N ¨ 1 RBER, BETTINA
Format:	Patent
Sprache:	chi ; eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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creator	JOENSEN LARS VIKESA JONAS N ¨ 1 RBER, BETTINA
description	The present invention relates to antisense oligonucleotide splicing modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for TDP-43 functional depleted conditions and medical indications. 本发明涉及肌动蛋白样6B(ACTL6B)的反义寡核苷酸剪接调节剂。这些反义寡核苷酸剪接调节剂与ACTL6B前体mRNA互补，诸如完全互补，并且能够在TDP-43耗竭的细胞中增加或恢复ACTL6B的表达，诸如用于TDP-43功能耗竭的病况和医学适应症。
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These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for TDP-43 functional depleted conditions and medical indications. 本发明涉及肌动蛋白样6B(ACTL6B)的反义寡核苷酸剪接调节剂。这些反义寡核苷酸剪接调节剂与ACTL6B前体mRNA互补，诸如完全互补，并且能够在TDP-43耗竭的细胞中增加或恢复ACTL6B的表达，诸如用于TDP-43功能耗竭的病况和医学适应症。</description><language>chi ; eng</language><subject>BEER ; BIOCHEMISTRY ; CHEMISTRY ; COMPOSITIONS THEREOF ; CULTURE MEDIA ; ENZYMOLOGY ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; 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=20240802&DB=EPODOC&CC=CN&NR=118434858A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,776,881,25542,76290</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20240802&DB=EPODOC&CC=CN&NR=118434858A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>JOENSEN LARS</creatorcontrib><creatorcontrib>VIKESA JONAS</creatorcontrib><creatorcontrib>N ¨ 1 RBER, BETTINA</creatorcontrib><title>Antisense oligonucleotides targeting ACTL6B</title><description>The present invention relates to antisense oligonucleotide splicing modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for TDP-43 functional depleted conditions and medical indications. 本发明涉及肌动蛋白样6B(ACTL6B)的反义寡核苷酸剪接调节剂。这些反义寡核苷酸剪接调节剂与ACTL6B前体mRNA互补，诸如完全互补，并且能够在TDP-43耗竭的细胞中增加或恢复ACTL6B的表达，诸如用于TDP-43功能耗竭的病况和医学适应症。</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>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</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>eNrjZNB2zCvJLE7NK05VyM_JTM_PK03OSc0vyUxJLVYoSSxKTy3JzEtXcHQO8TFz4mFgTUvMKU7lhdLcDIpuriHOHrqpBfnxqcUFicmpeakl8c5-hoYWJsYmFqYWjsbEqAEAYTIpSg</recordid><startdate>20240802</startdate><enddate>20240802</enddate><creator>JOENSEN LARS</creator><creator>VIKESA JONAS</creator><creator>N ¨ 1 RBER, BETTINA</creator><scope>EVB</scope></search><sort><creationdate>20240802</creationdate><title>Antisense oligonucleotides targeting ACTL6B</title><author>JOENSEN LARS ; VIKESA JONAS ; N ¨ 1 RBER, BETTINA</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_CN118434858A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>chi ; eng</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>METALLURGY</topic><topic>MICROBIOLOGY</topic><topic>MICROORGANISMS OR ENZYMES</topic><topic>MUTATION OR GENETIC ENGINEERING</topic><topic>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>SPIRITS</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>JOENSEN LARS</creatorcontrib><creatorcontrib>VIKESA JONAS</creatorcontrib><creatorcontrib>N ¨ 1 RBER, BETTINA</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>JOENSEN LARS</au><au>VIKESA JONAS</au><au>N ¨ 1 RBER, BETTINA</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Antisense oligonucleotides targeting ACTL6B</title><date>2024-08-02</date><risdate>2024</risdate><abstract>The present invention relates to antisense oligonucleotide splicing modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for TDP-43 functional depleted conditions and medical indications. 本发明涉及肌动蛋白样6B(ACTL6B)的反义寡核苷酸剪接调节剂。这些反义寡核苷酸剪接调节剂与ACTL6B前体mRNA互补，诸如完全互补，并且能够在TDP-43耗竭的细胞中增加或恢复ACTL6B的表达，诸如用于TDP-43功能耗竭的病况和医学适应症。</abstract><oa>free_for_read</oa></addata></record>
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title	Antisense oligonucleotides targeting ACTL6B
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