KR20230028375A - 원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법 - Google Patents

원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법 Download PDF

Info

Publication number
KR20230028375A
KR20230028375A KR1020237001270A KR20237001270A KR20230028375A KR 20230028375 A KR20230028375 A KR 20230028375A KR 1020237001270 A KR1020237001270 A KR 1020237001270A KR 20237001270 A KR20237001270 A KR 20237001270A KR 20230028375 A KR20230028375 A KR 20230028375A
Authority
KR
South Korea
Prior art keywords
ires
sequence
protein
nucleic acid
circular rna
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
KR1020237001270A
Other languages
English (en)
Korean (ko)
Inventor
하워드 와이. 창
춘-칸 첸
Original Assignee
더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티, 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 filed Critical 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
Publication of KR20230028375A publication Critical patent/KR20230028375A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/532Closed or circular
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
KR1020237001270A 2020-06-25 2021-06-25 원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법 Pending KR20230028375A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063043964P 2020-06-25 2020-06-25
US63/043,964 2020-06-25
US202163186507P 2021-05-10 2021-05-10
US63/186,507 2021-05-10
PCT/US2021/039127 WO2021263124A2 (en) 2020-06-25 2021-06-25 Genetic elements driving circular rna translation and methods of use

Publications (1)

Publication Number Publication Date
KR20230028375A true KR20230028375A (ko) 2023-02-28

Family

ID=79281918

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237001270A Pending KR20230028375A (ko) 2020-06-25 2021-06-25 원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법

Country Status (8)

Country Link
US (3) US11560567B2 (https=)
EP (1) EP4171631A4 (https=)
JP (1) JP2023532663A (https=)
KR (1) KR20230028375A (https=)
CN (1) CN116323945A (https=)
AU (1) AU2021297340A1 (https=)
CA (1) CA3180224A1 (https=)
WO (1) WO2021263124A2 (https=)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240299520A1 (en) * 2021-02-09 2024-09-12 Virginia Commonwealth University Mini circular rna therapeutics and vaccines and methods of use thereof
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
EP4436984A1 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Coronavirus immunogen compositions and their uses
CN118401544A (zh) 2021-11-24 2024-07-26 旗舰创业创新六公司 水痘-带状疱疹病毒免疫原组合物及其用途
KR20240118174A (ko) 2021-12-17 2024-08-02 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변성 조건 하에서의 원형 rna의 풍부화를 위한 방법
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
KR20240118881A (ko) 2021-12-23 2024-08-05 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 항푸소제닉 폴리펩티드를 인코딩하는 원형 폴리리보뉴클레오티드
CN119095967A (zh) * 2022-02-24 2024-12-06 普罗拜奥根股份公司 病毒样颗粒介导的环状rna递送
EP4493197A4 (en) * 2022-03-17 2026-03-18 Univ Leland Stanford Junior COMPOSITIONS AND METHODS FOR ENHANCED PROTEIN TRANSLATION FROM RECOMBED CIRCULAR RNA
US20250320519A1 (en) 2022-05-30 2025-10-16 Shanghai Circode Biomed Co., Ltd. Synthetic circular rna compositions and methods of use thereof
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状rna分子を調製するための組成物及び方法
US20260002223A1 (en) * 2022-06-27 2026-01-01 Wisconsin Alumni Research Foundation Universal method for parasite and eukaryotic endosymbiont identification
KR20240003761A (ko) * 2022-06-29 2024-01-09 서울대학교산학협력단 mRNA 번역 증가용 조절 엘리먼트를 스크리닝하는 방법,상기 방법에 따른 신규 조절 엘리먼트, 및 이의 용도
WO2024010028A1 (ja) * 2022-07-06 2024-01-11 国立大学法人京都大学 環状rna分子及びこれを用いた翻訳制御方法、翻訳活性化システム、並びに医薬組成物
CN115394376B (zh) * 2022-09-28 2023-04-18 奥明(杭州)生物医药有限公司 一种预测环状rna ires的方法
TW202434728A (zh) 2022-11-08 2024-09-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸的組成物和方法
KR20250122519A (ko) 2022-12-22 2025-08-13 젠스크립트 유에스에이 인크. 자가환형화 rna의 제조 방법
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
CN121057577A (zh) 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
CN121358494A (zh) 2023-03-15 2026-01-16 旗舰创业创新第六有限责任公司 免疫原性组合物及其用途
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024226621A1 (en) * 2023-04-25 2024-10-31 Ginkgo Bioworks, Inc. Novel internal ribosome entry site (ires) and intron sequences
EP4705481A2 (en) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024254546A2 (en) * 2023-06-08 2024-12-12 Cornell University Virus-like particles comprising circular mrna expression systems and methods of use thereof
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
WO2025034727A1 (en) * 2023-08-07 2025-02-13 Yale University Translational modulating elements for mrna molecules capped with 5'-cap analogs, mrna molecules including the same, and methods using the same
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025072615A1 (en) 2023-09-29 2025-04-03 Orbital Therapeutics, Inc. Improved methods of making rna by splinted ligation and compositions thereof
WO2025077734A1 (en) * 2023-10-09 2025-04-17 Shanghai Circode Biomed Co., Ltd. Constructs and methods for preparing circular rnas and uses thereof
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫受體抑制蛋白及相關方法
WO2025179198A1 (en) 2024-02-23 2025-08-28 Sail Biomedicines, Inc. Circular polyribonucleotides and unmodified linear rnas with reduced immunogenicity
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites
WO2026060273A1 (en) * 2024-09-13 2026-03-19 Shape Therapeutics Inc. 5' untranslated regions (utrs) for enhanced payload expression
WO2026072709A1 (en) 2024-09-25 2026-04-02 Orna Therapeutics, Inc. Chimeric antigen receptors targeting bcma

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5766903A (en) * 1995-08-23 1998-06-16 University Technology Corporation Circular RNA and uses thereof
US6869779B1 (en) * 1998-08-27 2005-03-22 Fuso Pharmaceutical Industries, Ltd. Nucleic acid sequence for potentiating the expression of useful gene and method therefor
DE10143238A1 (de) * 2001-09-04 2003-03-20 Icon Genetics Ag Identifizierung eukaryotischer interner Ribosomen-Eingangsstellen (IRES)-Elemente
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
US20050118625A1 (en) * 2003-10-02 2005-06-02 Mounts William M. Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases
WO2010084371A1 (en) 2009-01-26 2010-07-29 Mitoprod Novel circular interfering rna molecules
EP2531515B1 (en) * 2010-01-22 2015-10-21 Gen-Probe Incorporated Probes for detecting the presence of trochomonas vaginalis in a sample
DK2925866T3 (en) 2012-11-30 2018-10-29 Univ Aarhus CIRCULAR RNA FOR INHIBITING MICRO-RNA
EP2996697B1 (en) * 2013-05-15 2019-06-26 Robert Kruse Intracellular translation of circular rna
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
EP3374504B1 (en) * 2015-11-09 2025-03-19 CureVac SE Optimized nucleic acid molecules
WO2017222911A1 (en) 2016-06-20 2017-12-28 The Board Of Trustees Of The Leland Stanford Junior University Circular rnas and their use in immunomodulation
JP7385282B2 (ja) * 2017-11-07 2023-11-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 環状rna分子のための方法及び組成物
CN112399860B (zh) * 2018-06-06 2024-08-16 麻省理工学院 用于在真核细胞中翻译的环状rna
WO2020023655A1 (en) * 2018-07-24 2020-01-30 Flagship Pioneering, Inc. Compositions comprising circular polyribonucleotides and uses thereof
AU2019310448A1 (en) * 2018-07-24 2021-03-11 Mayo Foundation For Medical Education And Research Circularized engineered rna and methods
US20220362295A1 (en) 2019-04-22 2022-11-17 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using fusion proteins

Also Published As

Publication number Publication date
WO2021263124A3 (en) 2022-02-03
US11560567B2 (en) 2023-01-24
JP2023532663A (ja) 2023-07-31
CN116323945A (zh) 2023-06-23
US20230125935A1 (en) 2023-04-27
AU2021297340A1 (en) 2023-01-19
US20230407317A1 (en) 2023-12-21
EP4171631A4 (en) 2025-02-12
CA3180224A1 (en) 2021-12-30
US11685924B2 (en) 2023-06-27
WO2021263124A2 (en) 2021-12-30
US20220251578A1 (en) 2022-08-11
EP4171631A2 (en) 2023-05-03

Similar Documents

Publication Publication Date Title
US11685924B2 (en) Genetic elements driving circular RNA translation and methods of use
Liu et al. Circular RNAs: characterization, cellular roles, and applications
KR102405549B1 (ko) Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR20240024171A (ko) 재조합 원형 rna로부터 개선된 단백질 번역을 위한 조성물 및 방법
Liu et al. NAP-seq reveals multiple classes of structured noncoding RNAs with regulatory functions
CN116322791A (zh) 经修饰的功能性核酸分子
US20260015627A1 (en) Compositions and methods for improved protein translation from recombinant circular rnas
WO2022090733A1 (en) Functional nucleic acid molecules incorporating protein binding domains
Rapakko et al. Interplay between RNA‐protein interactions and RNA structures in gene regulation
JP7776924B2 (ja) 変異FUSの発現を選択的に抑制する修飾siRNA
EP4549591A1 (en) Method for screening regulatory element for increasing mrna translation, novel regulatory element resulting from method, and use thereof
CN103275978B (zh) 修饰的小干扰核酸及其制备方法
CN115038710B (zh) 基于结构的靶向rna发夹环的治疗剂的设计
Trevisan An RNA story, from chromatin to protein
Eufrásio et al. A conserved 3′ UTR short motif regulates gene expression in vertebrates
van Rensburg Investigating the coding region determinant binding protein-RNA interaction in vitro, in cells and in silico.
WO2011138787A1 (en) Identification of mrna-specific micrornas
Bak et al. Non-coding and Regulatory RNAs 157
Passoni Dinucleotide TG repeats and 5'splice site definition
NZ750917B2 (en) High throughput cell-based screening for aptamers

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902