CN109414449A - 利用和不利用可设计核酸酶编辑基因组的改进方法 - Google Patents

利用和不利用可设计核酸酶编辑基因组的改进方法 Download PDF

Info

Publication number
CN109414449A
CN109414449A CN201780038175.3A CN201780038175A CN109414449A CN 109414449 A CN109414449 A CN 109414449A CN 201780038175 A CN201780038175 A CN 201780038175A CN 109414449 A CN109414449 A CN 109414449A
Authority
CN
China
Prior art keywords
oligonucleotides
editor
target
dna
modification
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
CN201780038175.3A
Other languages
English (en)
Chinese (zh)
Inventor
托德·M·伍尔夫
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CN109414449A publication Critical patent/CN109414449A/zh
Pending legal-status Critical Current

Links

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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201780038175.3A 2016-05-06 2017-05-05 利用和不利用可设计核酸酶编辑基因组的改进方法 Pending CN109414449A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662333004P 2016-05-06 2016-05-06
US62/333,004 2016-05-06
US201662410487P 2016-10-20 2016-10-20
US62/410,487 2016-10-20
PCT/US2017/031381 WO2017193053A1 (en) 2016-05-06 2017-05-05 Improved methods for genome editing with and without programmable nucleases

Publications (1)

Publication Number Publication Date
CN109414449A true CN109414449A (zh) 2019-03-01

Family

ID=60203506

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780038175.3A Pending CN109414449A (zh) 2016-05-06 2017-05-05 利用和不利用可设计核酸酶编辑基因组的改进方法

Country Status (6)

Country Link
US (1) US20190300872A1 (https=)
EP (2) EP3452055A4 (https=)
JP (4) JP6872560B2 (https=)
CN (1) CN109414449A (https=)
CA (1) CA3022319A1 (https=)
WO (1) WO2017193053A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111378051A (zh) * 2020-03-25 2020-07-07 北京市农林科学院 Pe-p2引导编辑系统及其在基因组碱基编辑中的应用
CN113637670A (zh) * 2021-04-27 2021-11-12 中国药科大学 一种用于任意基因表达调控的组合物及方法

Families Citing this family (42)

* 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
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
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
US12359197B2 (en) * 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CA3022319A1 (en) * 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases
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
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
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
WO2019136459A1 (en) 2018-01-08 2019-07-11 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
EP3737743A1 (en) 2018-01-08 2020-11-18 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
WO2019204531A1 (en) * 2018-04-18 2019-10-24 Ligandal, Inc. Methods and compositions for genome editing
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
US20210079387A1 (en) * 2019-08-27 2021-03-18 The Jackson Laboratory Cleavage-resistant donor nucleic acids and methods of use
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20210247378A1 (en) * 2020-02-10 2021-08-12 Palogen, Inc. Nanopore device and methods of detecting and classifying charged particles using same
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
EP4107270A4 (en) * 2020-07-30 2024-08-14 Agency for Science, Technology and Research METHOD FOR STIMULATING THE PROLIFERATION OF A CELL
US20220056437A1 (en) * 2020-08-19 2022-02-24 Integrated Dna Technologies, Inc. Methods and compositions for inhibition of crispr re-cleavage events
US20250255986A1 (en) * 2021-11-01 2025-08-14 Christopher Bradley Dna revertase
WO2025101484A1 (en) 2023-11-06 2025-05-15 Iovance Biotherapeutics, Inc. Treatment of endometrial cancers with tumor infiltrating lymphocyte therapies

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150166980A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Fusions of cas9 domains and nucleic acid-editing domains

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3023787A1 (de) 1980-06-25 1982-01-21 Studiengesellschaft Kohle mbH, 4330 Mülheim Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
EP0452457B1 (en) 1989-11-03 1997-08-20 Vanderbilt University Method of in vivo delivery of functioning foreign genes
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US6936467B2 (en) 2000-03-27 2005-08-30 University Of Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
EP1268768A2 (en) 2000-03-27 2003-01-02 University Of Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8232383B2 (en) 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
DE60310944T3 (de) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2004094447A1 (ja) 2003-04-22 2004-11-04 Kyushu Tlo Company, Limited チオヌクレオシド-s-ニトロシル誘導体
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US8309356B2 (en) 2009-04-01 2012-11-13 Yale University Pseudocomplementary oligonucleotides for targeted gene therapy
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
WO2013166487A1 (en) 2012-05-04 2013-11-07 Yale University Highly penetrative nanocarriers for treatment of cns disease
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
RS54944B1 (sr) * 2012-07-12 2016-11-30 Proqr Therapeutics Ii Bv Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji
EP4136963B1 (en) * 2013-03-15 2026-01-21 Cibus US LLC Methods and compositions for increasing efficiency of targeted gene modification using oligonucleotide-mediated gene repair
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3115457B1 (en) * 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
KR102450868B1 (ko) * 2014-03-14 2022-10-06 시버스 유에스 엘엘씨 올리고뉴클레오타이드 매개 유전자 보수를 사용한 표적화된 유전자 변형의 효율을 증가시키기 위한 방법 및 조성물
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
SG11201701245QA (en) * 2014-08-27 2017-03-30 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
BR112017007770A2 (pt) * 2014-10-15 2018-01-16 Regeneron Pharma cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
US12359197B2 (en) * 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
JP7044373B2 (ja) * 2015-07-15 2022-03-30 ラトガース,ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
CA3022319A1 (en) * 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150166980A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Fusions of cas9 domains and nucleic acid-editing domains

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111378051A (zh) * 2020-03-25 2020-07-07 北京市农林科学院 Pe-p2引导编辑系统及其在基因组碱基编辑中的应用
CN111378051B (zh) * 2020-03-25 2022-03-01 北京市农林科学院 Pe-p2引导编辑系统及其在基因组碱基编辑中的应用
CN113637670A (zh) * 2021-04-27 2021-11-12 中国药科大学 一种用于任意基因表达调控的组合物及方法

Also Published As

Publication number Publication date
US20190300872A1 (en) 2019-10-03
EP4023228A1 (en) 2022-07-06
EP3452055A4 (en) 2019-11-06
JP2023011676A (ja) 2023-01-24
JP2019514405A (ja) 2019-06-06
JP2021119776A (ja) 2021-08-19
EP3452055A1 (en) 2019-03-13
CA3022319A1 (en) 2017-11-09
WO2017193053A1 (en) 2017-11-09
JP6872560B2 (ja) 2021-05-19
JP2024088642A (ja) 2024-07-02

Similar Documents

Publication Publication Date Title
CN109414449A (zh) 利用和不利用可设计核酸酶编辑基因组的改进方法
US12203072B2 (en) Chemically modified single-stranded rna-editing oligonucleotides
Wan et al. The medicinal chemistry of therapeutic oligonucleotides
KR102618864B1 (ko) 합성 가이드 분자, 그와 관련된 조성물 및 방법
EP3230445B1 (en) Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
US8658608B2 (en) Modified triple-helix forming oligonucleotides for targeted mutagenesis
EP3571300A1 (en) Oligonucleotide complexes for use in rna editing
EP3752611A1 (en) Antisense oligonucleotides for rna editing
WO2017220751A1 (en) Single-stranded rna-editing oligonucleotides
WO2022070107A1 (en) Materials and methods for treatment of amyotrophic lateral sclerosis
KR20260018069A (ko) Ptpn2 발현을 표적화하는 crispr 관련 방법 및 조성물
KR20260018068A (ko) Cd70 발현을 표적화하는 crispr 관련 방법 및 조성물
WO2024256620A1 (en) Antisense oligonucleotides for the treatment of neurodegenerative disease
CN121219418A (zh) 靶向fl1-1表达的crispr相关方法和组合物

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
RJ01 Rejection of invention patent application after publication
RJ01 Rejection of invention patent application after publication

Application publication date: 20190301