Application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease
The invention discloses an application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease, and finds for the first time that expression conversion of gamma-globin to beta-globin can be completely reversed by destroying interaction between...
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| creator | LIU DEPEI ZHANG ZHUQIN WANG NANYU XIONG MIN YANG KE |
| description | The invention discloses an application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease, and finds for the first time that expression conversion of gamma-globin to beta-globin can be completely reversed by destroying interaction between HBB and LCR and simultaneously relieving apparent silencing on HBG. The discovery has an important clinical application prospect in the field of beta-hemoglobin disease gene therapy.
本发明公开了同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用，本发明首次发现破坏HBB与LCR间的相互作用的同时解除HBG上的表观沉默可以完全逆转γ-向β-珠蛋白表达转换，该发现在β-血红蛋白病基因治疗领域具有重要的临床应用前景。 |
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本发明公开了同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用，本发明首次发现破坏HBB与LCR间的相互作用的同时解除HBG上的表观沉默可以完全逆转γ-向β-珠蛋白表达转换，该发现在β-血红蛋白病基因治疗领域具有重要的临床应用前景。</description><language>chi ; eng</language><subject>HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; 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=20240123&DB=EPODOC&CC=CN&NR=117427169A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25564,76547</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20240123&DB=EPODOC&CC=CN&NR=117427169A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>LIU DEPEI</creatorcontrib><creatorcontrib>ZHANG ZHUQIN</creatorcontrib><creatorcontrib>WANG NANYU</creatorcontrib><creatorcontrib>XIONG MIN</creatorcontrib><creatorcontrib>YANG KE</creatorcontrib><title>Application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease</title><description>The invention discloses an application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease, and finds for the first time that expression conversion of gamma-globin to beta-globin can be completely reversed by destroying interaction between HBB and LCR and simultaneously relieving apparent silencing on HBG. The discovery has an important clinical application prospect in the field of beta-hemoglobin disease gene therapy.
本发明公开了同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用，本发明首次发现破坏HBB与LCR间的相互作用的同时解除HBG上的表观沉默可以完全逆转γ-向β-珠蛋白表达转换，该发现在β-血红蛋白病基因治疗领域具有重要的临床应用前景。</description><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</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>eNqNjDEOgkAQRWksjHqH9QAUqJFYAlGprOzJsAy4ye7Mhh0K4uVdjAew-sn77_918i68t0aDGCbFvQrGTVaAkKdgZ9VhkJFnQ4Oqy1Jpdh7FfGWgLrK7Au9hRJI4tUh6UQ0pGRHELTietiiQvtDxYLmNZWcCQsBtsurBBtz9cpPsb9dnVafoucHgQSOhNNUjy_LTIc_Ol-L4j_MB2adHqg</recordid><startdate>20240123</startdate><enddate>20240123</enddate><creator>LIU DEPEI</creator><creator>ZHANG ZHUQIN</creator><creator>WANG NANYU</creator><creator>XIONG MIN</creator><creator>YANG KE</creator><scope>EVB</scope></search><sort><creationdate>20240123</creationdate><title>Application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease</title><author>LIU DEPEI ; ZHANG ZHUQIN ; WANG NANYU ; XIONG MIN ; YANG KE</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_CN117427169A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>chi ; eng</language><creationdate>2024</creationdate><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</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>LIU DEPEI</creatorcontrib><creatorcontrib>ZHANG ZHUQIN</creatorcontrib><creatorcontrib>WANG NANYU</creatorcontrib><creatorcontrib>XIONG MIN</creatorcontrib><creatorcontrib>YANG KE</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>LIU DEPEI</au><au>ZHANG ZHUQIN</au><au>WANG NANYU</au><au>XIONG MIN</au><au>YANG KE</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease</title><date>2024-01-23</date><risdate>2024</risdate><abstract>The invention discloses an application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease, and finds for the first time that expression conversion of gamma-globin to beta-globin can be completely reversed by destroying interaction between HBB and LCR and simultaneously relieving apparent silencing on HBG. The discovery has an important clinical application prospect in the field of beta-hemoglobin disease gene therapy.
本发明公开了同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用，本发明首次发现破坏HBB与LCR间的相互作用的同时解除HBG上的表观沉默可以完全逆转γ-向β-珠蛋白表达转换，该发现在β-血红蛋白病基因治疗领域具有重要的临床应用前景。</abstract><oa>free_for_read</oa></addata></record> |
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| title | Application of simultaneously destroying HBB competition and HBG apparent silencing in treatment of beta-hemoglobin disease |
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