DK3204399T3 - Lange poly (a)-plasmider og fremgangsmåder til indføring af lange poly (a)-sekvenser i plasmidet - Google Patents

Lange poly (a)-plasmider og fremgangsmåder til indføring af lange poly (a)-sekvenser i plasmidet Download PDF

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Publication number
DK3204399T3
DK3204399T3 DK15848462.6T DK15848462T DK3204399T3 DK 3204399 T3 DK3204399 T3 DK 3204399T3 DK 15848462 T DK15848462 T DK 15848462T DK 3204399 T3 DK3204399 T3 DK 3204399T3
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long poly
plasmids
plasmid
sequences
methods
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DK15848462.6T
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English (en)
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Andrew M Scharenberg
Kyle Jacoby
Alexandra E Grier
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Seattle Childrens Hospital Dba Seattle Childrens Res Inst
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0047Sonopheresis, i.e. ultrasonically-enhanced transdermal delivery, electroporation of a pharmacologically active agent
    • 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
    • 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
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK15848462.6T 2014-10-09 2015-10-08 Lange poly (a)-plasmider og fremgangsmåder til indføring af lange poly (a)-sekvenser i plasmidet DK3204399T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462062098P 2014-10-09 2014-10-09
US201562161107P 2015-05-13 2015-05-13
PCT/US2015/054780 WO2016057850A1 (en) 2014-10-09 2015-10-08 Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid

Publications (1)

Publication Number Publication Date
DK3204399T3 true DK3204399T3 (da) 2025-02-24

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DK15848462.6T DK3204399T3 (da) 2014-10-09 2015-10-08 Lange poly (a)-plasmider og fremgangsmåder til indføring af lange poly (a)-sekvenser i plasmidet

Country Status (6)

Country Link
US (3) US9943612B2 (da)
EP (2) EP4553081A1 (da)
AU (2) AU2015330775B2 (da)
DK (1) DK3204399T3 (da)
ES (1) ES3015369T3 (da)
WO (1) WO2016057850A1 (da)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP4282419A1 (en) 2012-12-20 2023-11-29 Purdue Research Foundation Chimeric antigen receptor-expressing t cells as anti-cancer therapeutics
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US20170335331A1 (en) 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
CA2985650A1 (en) * 2015-05-13 2016-11-17 Seattle Children's Hospital (dba Seattle Children's Research Institute) Enhancing endonuclease based gene editing in primary cells
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3439675A4 (en) 2016-04-08 2019-12-18 Purdue Research Foundation METHOD AND COMPOSITIONS FOR CAR-T CELL THERAPY
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018160622A1 (en) 2017-02-28 2018-09-07 Endocyte, Inc. Compositions and methods for car t cell therapy
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
CA3072960A1 (en) * 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11311576B2 (en) 2018-01-22 2022-04-26 Seattle Children's Hospital Methods of use for CAR T cells
AU2019225174B2 (en) 2018-02-23 2025-11-20 Endocyte, Inc. Sequencing method for CAR T cell therapy
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
US20230348902A1 (en) * 2020-08-10 2023-11-02 Integral Molecular, Inc. Methods and compositions for making antibody libraries and antibodies isolated from the same
US11918643B2 (en) 2020-12-22 2024-03-05 CureVac SE RNA vaccine against SARS-CoV-2 variants
US20240084285A1 (en) * 2021-01-22 2024-03-14 Nanjing GenScript Biotech Co., Ltd. Method for producing target dna sequence and cloning vector
US20240102065A1 (en) 2021-01-27 2024-03-28 CureVac SE Method of reducing the immunostimulatory properties of in vitro transcribed rna
NL2027654B1 (en) 2021-02-08 2022-09-09 Nutcracker Therapeutics Inc Methods for manufacturing a syntetic template
CN116004682B (zh) * 2022-08-19 2026-01-16 中国人民解放军军事科学院军事医学研究院 一种快速无痕制备含长多聚腺嘌呤的mRNA的方法及应用
CN117701602A (zh) * 2022-08-26 2024-03-15 深圳瑞吉生物科技有限公司 在宿主细胞中稳定存在的多段化PolyA多核苷酸及其应用
EP4608442A1 (en) 2022-10-28 2025-09-03 GlaxoSmithKline Biologicals S.A. Nucleic acid based vaccine
CN119799695A (zh) * 2023-10-10 2025-04-11 深圳瑞吉生物科技有限公司 提高mRNA稳定性以及蛋白表达的多元polyA多核苷酸及其应用

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030083272A1 (en) * 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
US6130040A (en) 1998-01-08 2000-10-10 Lin; Shi-Lung Method for generating directly covalent bonding between interstrand nucleotides
US20010000077A1 (en) 1998-02-03 2001-03-29 Engelhardt Dean L. Novel process, construct and conjugate for producing multiple nucleic acid copies
AU766146B2 (en) * 1998-05-26 2003-10-09 University Of Medicine And Dentistry Of New Jersey System for reproducing and modulating stability and turnover of RNA molecules
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
ATE549408T1 (de) * 2006-01-12 2012-03-15 Lucigen Corp Lineare vektoren, wirtszellen und klonverfahren
US7723103B2 (en) * 2007-01-08 2010-05-25 Lucigen Corporation Vectors, kits and methods for cloning DNA
DE102007003321A1 (de) * 2007-01-17 2008-08-21 Mbb International Group Ag Verschlusseinrichtung für einen mit Druckgas befüllbaren Druckbehälter eines Kaltgasgenerators
WO2008097926A2 (en) * 2007-02-02 2008-08-14 Yale University Transient transfection with rna
WO2013123559A1 (en) 2012-02-22 2013-08-29 Monash University Rna tagging method
WO2013143700A2 (en) * 2012-03-27 2013-10-03 Curevac Gmbh Artificial nucleic acid molecules comprising a 5'top utr
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US9650406B2 (en) 2013-02-28 2017-05-16 Centrillion Technology Holdings Corporation Reversible terminator molecules and methods of their use
WO2016005004A1 (en) * 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
CA3072960A1 (en) * 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)

Also Published As

Publication number Publication date
US9943612B2 (en) 2018-04-17
US20180272007A1 (en) 2018-09-27
EP3204399B1 (en) 2025-01-22
ES3015369T3 (en) 2025-05-05
WO2016057850A1 (en) 2016-04-14
CA2963840A1 (en) 2016-04-14
US11400169B2 (en) 2022-08-02
AU2015330775A1 (en) 2017-05-18
EP3204399A4 (en) 2018-04-25
EP4553081A1 (en) 2025-05-14
EP3204399A1 (en) 2017-08-16
AU2021203655A1 (en) 2021-07-01
US20160243258A1 (en) 2016-08-25
AU2015330775B2 (en) 2021-06-24
US20230054471A1 (en) 2023-02-23

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