PAH 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
본원에서는, 세포에서 페닐알라닌 히드록실라아제(PAH) 유전자 기능을 복원할 수 있는 재조합 아데노-연관 바이러스(AAV) 조성물, 및 PAH 유전자 기능의 감소와 연관된 장애(예를 들어, PKU)를 치료하기 위해 이를 사용하는 방법이 제공된다. 또한, 아데노-관련 바이러스 조성물을 제조하기 위한 핵산, 벡터, 패키징 시스템, 및 방법이 제공된다. Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanin...
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| creator | ST. MARTIN THIA BABOVAL WRIGHT JASON BOKE SEYMOUR ALBERT BARNES SOOKIASIAN DANIELLE LAUREN FRANCONE OMAR |
| description | 본원에서는, 세포에서 페닐알라닌 히드록실라아제(PAH) 유전자 기능을 복원할 수 있는 재조합 아데노-연관 바이러스(AAV) 조성물, 및 PAH 유전자 기능의 감소와 연관된 장애(예를 들어, PKU)를 치료하기 위해 이를 사용하는 방법이 제공된다. 또한, 아데노-관련 바이러스 조성물을 제조하기 위한 핵산, 벡터, 패키징 시스템, 및 방법이 제공된다.
Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions. |
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Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.</description><language>kor</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 ; ORGANIC CHEMISTRY ; PEPTIDES ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; SPIRITS ; VINEGAR ; WINE</subject><creationdate>2023</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=20230303&DB=EPODOC&CC=KR&NR=20230029650A$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25563,76418</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20230303&DB=EPODOC&CC=KR&NR=20230029650A$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>ST. MARTIN THIA BABOVAL</creatorcontrib><creatorcontrib>WRIGHT JASON BOKE</creatorcontrib><creatorcontrib>SEYMOUR ALBERT BARNES</creatorcontrib><creatorcontrib>SOOKIASIAN DANIELLE LAUREN</creatorcontrib><creatorcontrib>FRANCONE OMAR</creatorcontrib><title>PAH 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법</title><description>본원에서는, 세포에서 페닐알라닌 히드록실라아제(PAH) 유전자 기능을 복원할 수 있는 재조합 아데노-연관 바이러스(AAV) 조성물, 및 PAH 유전자 기능의 감소와 연관된 장애(예를 들어, PKU)를 치료하기 위해 이를 사용하는 방법이 제공된다. 또한, 아데노-관련 바이러스 조성물을 제조하기 위한 핵산, 벡터, 패키징 시스템, 및 방법이 제공된다.
Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.</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>ORGANIC CHEMISTRY</subject><subject>PEPTIDES</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</subject><subject>SPIRITS</subject><subject>VINEGAR</subject><subject>WINE</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2023</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZMgJcPRQeDNnwZsFLW_mTVB4tWPD666lb-a2KLzevPXV1jVvp84ACgEVtLydOkfhzdSW170bXrfu0H0zfcOrLQ0KrzdMeTN3y-v5a950LVF4s3DDm5aNr9fsAQr3KwDF38ydofCmac2bWSuBIitfb5rKw8CalphTnMoLpbkZlN1cQ5w9dFML8uNTiwsSk1PzUkvivYOMDIyMDQyMLM1MDRyNiVMFALNEX-g</recordid><startdate>20230303</startdate><enddate>20230303</enddate><creator>ST. MARTIN THIA BABOVAL</creator><creator>WRIGHT JASON BOKE</creator><creator>SEYMOUR ALBERT BARNES</creator><creator>SOOKIASIAN DANIELLE LAUREN</creator><creator>FRANCONE OMAR</creator><scope>EVB</scope></search><sort><creationdate>20230303</creationdate><title>PAH 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법</title><author>ST. MARTIN THIA BABOVAL ; WRIGHT JASON BOKE ; SEYMOUR ALBERT BARNES ; SOOKIASIAN DANIELLE LAUREN ; FRANCONE OMAR</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_KR20230029650A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>kor</language><creationdate>2023</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>ORGANIC CHEMISTRY</topic><topic>PEPTIDES</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>SPIRITS</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>ST. MARTIN THIA BABOVAL</creatorcontrib><creatorcontrib>WRIGHT JASON BOKE</creatorcontrib><creatorcontrib>SEYMOUR ALBERT BARNES</creatorcontrib><creatorcontrib>SOOKIASIAN DANIELLE LAUREN</creatorcontrib><creatorcontrib>FRANCONE OMAR</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>ST. MARTIN THIA BABOVAL</au><au>WRIGHT JASON BOKE</au><au>SEYMOUR ALBERT BARNES</au><au>SOOKIASIAN DANIELLE LAUREN</au><au>FRANCONE OMAR</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>PAH 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법</title><date>2023-03-03</date><risdate>2023</risdate><abstract>본원에서는, 세포에서 페닐알라닌 히드록실라아제(PAH) 유전자 기능을 복원할 수 있는 재조합 아데노-연관 바이러스(AAV) 조성물, 및 PAH 유전자 기능의 감소와 연관된 장애(예를 들어, PKU)를 치료하기 위해 이를 사용하는 방법이 제공된다. 또한, 아데노-관련 바이러스 조성물을 제조하기 위한 핵산, 벡터, 패키징 시스템, 및 방법이 제공된다.
Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.</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 ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE |
| title | PAH 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 |
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