JP2023527875A5 - - Google Patents

Info

Publication number
JP2023527875A5
JP2023527875A5 JP2022573670A JP2022573670A JP2023527875A5 JP 2023527875 A5 JP2023527875 A5 JP 2023527875A5 JP 2022573670 A JP2022573670 A JP 2022573670A JP 2022573670 A JP2022573670 A JP 2022573670A JP 2023527875 A5 JP2023527875 A5 JP 2023527875A5
Authority
JP
Japan
Prior art keywords
seq
mrna
amino acid
polypeptide
acid sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022573670A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023527875A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/035154 external-priority patent/WO2021247507A1/en
Publication of JP2023527875A publication Critical patent/JP2023527875A/ja
Publication of JP2023527875A5 publication Critical patent/JP2023527875A5/ja
Pending legal-status Critical Current

Links

JP2022573670A 2020-06-01 2021-06-01 フェニルアラニンヒドロキシラーゼバリアント及びその使用 Pending JP2023527875A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063032875P 2020-06-01 2020-06-01
US63/032,875 2020-06-01
PCT/US2021/035154 WO2021247507A1 (en) 2020-06-01 2021-06-01 Phenylalanine hydroxylase variants and uses thereof

Publications (2)

Publication Number Publication Date
JP2023527875A JP2023527875A (ja) 2023-06-30
JP2023527875A5 true JP2023527875A5 (enExample) 2024-06-10

Family

ID=76601819

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022573670A Pending JP2023527875A (ja) 2020-06-01 2021-06-01 フェニルアラニンヒドロキシラーゼバリアント及びその使用

Country Status (6)

Country Link
US (1) US20230235298A1 (enExample)
EP (1) EP4158005A1 (enExample)
JP (1) JP2023527875A (enExample)
AU (1) AU2021285812A1 (enExample)
CA (1) CA3184474A1 (enExample)
WO (1) WO2021247507A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
MX2023003805A (es) 2020-10-01 2023-04-12 Genzyme Corp Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado.
WO2023069948A1 (en) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
US20250313830A1 (en) * 2022-03-25 2025-10-09 Modernatx, Inc. Messenger ribonucleic acids with extended half-life
WO2025059290A1 (en) * 2023-09-14 2025-03-20 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria

Family Cites Families (132)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
US5563250A (en) 1987-12-02 1996-10-08 Neorx Corporation Cleavable conjugates for the delivery and release of agents in native form
US5505931A (en) 1993-03-04 1996-04-09 The Dow Chemical Company Acid cleavable compounds, their preparation and use as bifunctional acid-labile crosslinking agents
GB2284210B (en) 1993-11-26 1997-11-12 British Tech Group Translational enhancer DNA from a kinesin gene
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
GB9518395D0 (en) 1995-09-08 1995-11-08 Therexsys Ltd Plasmid stabilization
US6630351B1 (en) 1999-06-07 2003-10-07 Mirus Corporation Compositions and methods for drug delivery using pH sensitive molecules
US6004573A (en) 1997-10-03 1999-12-21 Macromed, Inc. Biodegradable low molecular weight triblock poly(lactide-co-glycolide) polyethylene glycol copolymers having reverse thermal gelation properties
AU1819499A (en) 1997-12-12 1999-06-28 Samyang Corporation Positively-charged poly{alpha-(omega-aminoalkyl)glycolic acid} for the delivery of a bioactive agent via tissue and cellular uptake
US6517869B1 (en) 1997-12-12 2003-02-11 Expression Genetics, Inc. Positively charged poly(alpha-(omega-aminoalkyl)lycolic acid) for the delivery of a bioactive agent via tissue and cellular uptake
DE69941447D1 (de) 1998-01-05 2009-11-05 Univ Washington Erhöhter transport unter benutzung membranzerstörender stoffe
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
DE69939740D1 (de) 1998-05-20 2008-11-27 Expression Genetics Inc Laktose oder galaktose-polyethylen glykol-gepfropfte poly-l-lysin als genträger
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
EP1117720A4 (en) 1998-07-13 2001-11-14 Expression Genetics Inc POLYESTER POLY-L-LYSINE ANALOG USED AS A BIODEGRADABLE SOLUBLE GENE CONVEYOR
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
US7737108B1 (en) 2000-01-07 2010-06-15 University Of Washington Enhanced transport using membrane disruptive agents
US6696038B1 (en) 2000-09-14 2004-02-24 Expression Genetics, Inc. Cationic lipopolymer as biocompatible gene delivery agent
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US6897196B1 (en) 2001-02-07 2005-05-24 The Regents Of The University Of California pH sensitive lipids based on ortho ester linkers, composition and method
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US6586524B2 (en) 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
EP1412065A2 (en) 2001-07-27 2004-04-28 President And Fellows Of Harvard College Laminar mixing apparatus and methods
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
WO2003092665A2 (en) 2002-05-02 2003-11-13 Massachusetts Eye And Ear Infirmary Ocular drug delivery systems and use thereof
WO2004020605A2 (en) 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
US7179456B2 (en) 2003-04-30 2007-02-20 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Use of parvovirus for brain tumor therapy
US7883720B2 (en) 2003-07-09 2011-02-08 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US8372966B2 (en) 2003-12-19 2013-02-12 University Of Cincinnati Oligonucleotide decoys and methods of use
CA2563533C (en) 2004-04-15 2013-10-01 Shmuel A. Ben-Sasson Compositions capable of facilitating penetration across a biological barrier
US8057821B2 (en) 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
WO2006063249A2 (en) 2004-12-10 2006-06-15 Justin Hanes Functionalized poly (ether-anhydride) block copolymers
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006110776A2 (en) 2005-04-12 2006-10-19 Nektar Therapeutics Al, Corporation Polyethylene glycol cojugates of antimicrobial agents
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
CA2613442C (en) 2005-06-30 2016-08-23 Archemix Corp. Materials and methods for the generation of fully 2'-modified nucleic acid transcripts
JP5135220B2 (ja) 2005-09-01 2013-02-06 ノバルティス ヴァクシンズ アンド ダイアグノスティクス ゲーエムベーハー アンド カンパニー カーゲー 血清群c髄膜炎菌を含む複数ワクチン接種
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
AU2006325030B2 (en) 2005-12-16 2012-07-26 Cellectis Cell penetrating peptide conjugates for delivering nucleic acids into cells
KR100859972B1 (ko) 2006-02-20 2008-09-25 이화여자대학교 산학협력단 막투과 단백질 도메인 펩타이드
US8309680B2 (en) 2006-02-21 2012-11-13 Nektar Therapeutics Segmented degradable polymers and conjugates made therefrom
US8313777B2 (en) 2006-10-05 2012-11-20 The Johns Hopkins University Water-dispersible oral, parenteral, and topical formulations for poorly water soluble drugs using smart polymeric nanoparticles
EP2500015A1 (en) 2006-12-05 2012-09-19 Landec Corporation Delivery of drugs
AU2007339280B2 (en) 2006-12-21 2013-12-05 Stryker Corporation Sustained-release formulations comprising crystals, macromolecular gels, and particulate suspensions of biologic agents
WO2008078180A2 (en) 2006-12-22 2008-07-03 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
JP5606738B2 (ja) 2006-12-27 2014-10-15 ウェルズ ファーゴ バンク ナショナル アソシエイション 解離可能な連結を有する第ix因子部分−ポリマー共役体
CA2689042A1 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
HUE040417T2 (hu) 2007-05-04 2019-03-28 Marina Biotech Inc Aminosavlipidek és alkalmazásuk
CA2715078C (en) 2007-09-26 2019-07-23 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
EP2436376B1 (en) 2007-09-28 2014-07-09 BIND Therapeutics, Inc. Cancer cell targeting using nanoparticles
WO2009114854A1 (en) 2008-03-14 2009-09-17 Egen, Inc. Biodegradable cross-linked branched poly (alkylene imines)
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
JP2011525180A (ja) 2008-06-16 2011-09-15 バインド バイオサイエンシズ インコーポレイテッド 治療的標的化ナノ粒子の製作に用いるためのジブロックコポリマーで官能化された標的薬の製造方法
WO2010005726A2 (en) 2008-06-16 2010-01-14 Bind Biosciences Inc. Therapeutic polymeric nanoparticles with mtor inhibitors and methods of making and using same
US8318211B2 (en) 2008-06-16 2012-11-27 Bind Biosciences, Inc. Therapeutic polymeric nanoparticles comprising vinca alkaloids and methods of making and using same
EA020954B1 (ru) 2008-06-16 2015-03-31 Бинд Терапьютикс, Инк. Загруженные лекарственным средством полимерные наночастицы, фармацевтическая композиция и способ лечения рака
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
JP2012512175A (ja) 2008-12-15 2012-05-31 バインド バイオサイエンシズ インコーポレイテッド 治療薬を徐放するための長時間循環性ナノ粒子
WO2010087791A1 (en) 2009-01-27 2010-08-05 Utc Power Corporation Distributively cooled, integrated water-gas shift reactor and vaporizer
US8460696B2 (en) 2009-03-20 2013-06-11 Egen, Inc. Polyamine derivatives
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
US9415113B2 (en) 2009-11-18 2016-08-16 University Of Washington Targeting monomers and polymers having targeting blocks
EP2515942B1 (en) 2009-12-15 2020-02-12 Pfizer Inc. Therapeutic polymeric nanoparticle compositions with high glass transition temperature or high molecular weight copolymers
EA201290497A1 (ru) 2009-12-15 2013-01-30 Байнд Байосайенсиз, Инк. Терапевтические полимерные наночастицы, включающие кортикостероиды, и способы получения таковых
WO2011084521A2 (en) 2009-12-15 2011-07-14 Bind Biosciences, Inc. Therapeutic polymeric nanoparticles comprising epothilone and methods of making and using same
US20130231287A1 (en) 2010-02-25 2013-09-05 Parimala Nacharaju Pegylated albumin polymers and uses thereof
US20130037977A1 (en) 2010-04-08 2013-02-14 Paul A. Burke Preparation of Lipid Nanoparticles
US20110250264A1 (en) 2010-04-09 2011-10-13 Pacira Pharmaceuticals, Inc. Method for formulating large diameter synthetic membrane vesicles
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
EP2397146A1 (en) 2010-06-11 2011-12-21 Deutsches Krebsforschungszentrum Parvovirus for treatment of tumours by intranasal application
RU2556800C2 (ru) 2010-06-14 2015-07-20 Ф.Хоффманн-Ля Рош Аг Пептиды, проникающие в клетки, и их применения
WO2011163483A2 (en) 2010-06-25 2011-12-29 Massachusetts Institute Of Technology Polymers for biomaterials and therapeutics
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
NZ606591A (en) 2010-07-06 2015-02-27 Novartis Ag Cationic oil-in-water emulsions
MX342608B (es) 2010-07-06 2016-10-06 Novartis Ag * Particulas de suministro similares a viriones para moleculas de arn de autorreplicacion.
WO2012012772A2 (en) 2010-07-22 2012-01-26 The Johns Hopkins University Drug eluting hydrogels for catheter delivery
KR101761388B1 (ko) 2010-07-30 2017-07-25 큐어백 아게 트랜스펙션 및 면역 자극을 위한 이황화-크로스링크된 양이온 성분 및 핵산의 복합체
WO2012021516A2 (en) 2010-08-09 2012-02-16 The Trustees Of The University Of Pennsylvania Nanoparticle-oligonucletide hybrid structures and methods of use thereof
EP2606072A4 (en) 2010-08-19 2016-04-20 Peg Biosciences Inc SYNERGISTIC BIOMOLECULAR POLYMER CONJUGATES
MX2013002332A (es) 2010-08-31 2013-03-18 Novartis Ag Lipidos apropiados para suministro liposomal del arn que codifica la proteina.
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
SI4043040T1 (sl) 2010-08-31 2023-04-28 Glaxosmithkline Biologicals Sa Mali liposomi za dostavo imunogen-kodirajoče RNA
US10307372B2 (en) 2010-09-10 2019-06-04 The Johns Hopkins University Rapid diffusion of large polymeric nanoparticles in the mammalian brain
EP2618846A1 (en) 2010-09-24 2013-07-31 Mallinckrodt LLC Aptamer conjugates for targeting of therapeutic and/or diagnostic nanocarriers
US20150056300A1 (en) 2010-10-22 2015-02-26 Bind Therapeutics, Inc. Therapeutic nanoparticles with high molecular weight copolymers
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
WO2012109121A1 (en) 2011-02-07 2012-08-16 Purdue Research Foundation Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2489371A1 (en) 2011-02-18 2012-08-22 Instituto Nacional de Investigacion y Tecnologia Agraria y Alimentaria Carrier peptides for drug delivery
US8846850B2 (en) 2011-02-22 2014-09-30 Rutgers, The State University Of New Jersey Amphiphilic macromolecules for nucleic acid delivery
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
JP2014527071A (ja) 2011-08-31 2014-10-09 マリンクロッド エルエルシー H−ホスホネートによるナノ粒子pegの改変
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND METHOD OF APPLICATION THEREFOR
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
WO2013082111A2 (en) 2011-11-29 2013-06-06 The University Of North Carolina At Chapel Hill Geometrically engineered particles and methods for modulating macrophage or immune responses
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
AU2012347605B2 (en) 2011-12-07 2017-09-21 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
EP2787977A4 (en) 2011-12-09 2015-05-06 Univ California LIPOSOMAL ACTIVE INVERTING
WO2013106072A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013106073A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013106086A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013105101A1 (en) 2012-01-13 2013-07-18 Department Of Biotechnology Solid lipid nanoparticles entrapping hydrophilic/ amphiphilic drug and a process for preparing the same
EP2804632B1 (en) 2012-01-19 2019-09-18 The Johns Hopkins University Nanoparticle formulations with enhanced mucosal penetration
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
JP6356614B2 (ja) 2012-02-17 2018-07-11 ユニバーシティ・オブ・ジョージア・リサーチ・ファウンデイション・インコーポレイテッド 薬剤のミトコンドリア輸送のためのナノ粒子
WO2013123523A1 (en) 2012-02-19 2013-08-22 Nvigen, Inc. Uses of porous nanostructure in delivery
WO2013124867A1 (en) 2012-02-21 2013-08-29 Amrita Vishwa Vidyapeetham University Polymer - polymer or polymer - protein core - shell nano medicine loaded with multiple drug molecules
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
CA2876860A1 (en) 2012-05-30 2013-12-05 Baylor College Of Medicine Supercoiled minivectors as a tool for dna repair, alteration and replacement
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
CN104342453A (zh) 2013-08-06 2015-02-11 深圳先进技术研究院 含基因工程抗体基因表达盒的微环dna重组母质粒、含该表达盒的微环dna及应用
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
EP3143153A4 (en) * 2014-05-16 2018-01-03 The University Of Georgia Research Foundation, Inc. Microbial approach for the production of 5-hydroxytryptophan
WO2016120186A1 (en) 2015-01-27 2016-08-04 F. Hoffmann-La Roche Ag Recombinant hbv cccdna, the method to generate thereof and the use thereof
EP3350333B2 (en) 2015-09-17 2025-08-06 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
WO2017066797A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Trinucleotide mrna cap analogs
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
WO2017167866A1 (en) * 2016-03-31 2017-10-05 Danmarks Tekniske Universitet Optimized microbial cells for production of melatonin and other compounds
WO2018167621A1 (en) * 2017-03-16 2018-09-20 Pfizer Inc. Tyrosine prototrophy
US11939601B2 (en) * 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy

Similar Documents

Publication Publication Date Title
JP2023527875A5 (enExample)
AU2020313143B2 (en) Targeted RNA editing by leveraging endogenous adar using engineered RNAs
JP2022544412A5 (enExample)
CN108929870B (zh) 抑制HBV的siRNA分子及其应用
WO2024097874A1 (en) Chemical stability of mrna
EP3332007A2 (en) Rnai therapy for hepatitis b virus infection
JP2015519047A5 (enExample)
TW201408309A (zh) 用於治療b型肝炎及d型肝炎感染之方法
JP2002529066A5 (enExample)
CA3159799A1 (en) Engineering circular guide rnas
WO2023242425A1 (en) Compositions and methods for circular rna affinity purification
TW201408308A (zh) 寡核苷酸螯合複合物-多肽組合物及方法
JP2022553375A5 (enExample)
CN114276419B (zh) 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
US20260009042A1 (en) RNA Having Lariat-Cap Structure For Improving Intracellular Stability And Biogenesis Of mRNA, And Use Thereof
JP2023506842A5 (enExample)
JP2025510777A5 (enExample)
JP2023510784A5 (enExample)
JP2024529108A5 (enExample)
JP2024501022A5 (enExample)
JP2025508841A5 (enExample)
WO2024235283A1 (en) Method of modulating rna splicing
WO2024230802A1 (en) Engineered liver-specific core promoters and their applications
CN109731095A (zh) 一种用于预防或治疗乙型肝炎病毒感染的药物及制备方法和应用
WO2026007374A1 (zh) 靶向乙型肝炎病毒基因的工程化基因转录阻抑工具及其用途