HK1258288A1 - 核酸酶非依賴性靶向基因編輯平台及其用途 - Google Patents

核酸酶非依賴性靶向基因編輯平台及其用途

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Publication number
HK1258288A1
HK1258288A1 HK19100652.8A HK19100652A HK1258288A1 HK 1258288 A1 HK1258288 A1 HK 1258288A1 HK 19100652 A HK19100652 A HK 19100652A HK 1258288 A1 HK1258288 A1 HK 1258288A1
Authority
HK
Hong Kong
Prior art keywords
nuclease
gene editing
targeted gene
editing platform
independent targeted
Prior art date
Application number
HK19100652.8A
Other languages
English (en)
Inventor
金晟侃
J-C‧科蘭特斯
Original Assignee
新澤西魯特格斯州立大學
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=57758208&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1258288(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 新澤西魯特格斯州立大學 filed Critical 新澤西魯特格斯州立大學
Publication of HK1258288A1 publication Critical patent/HK1258288A1/zh

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
HK19100652.8A 2015-07-15 2019-01-15 核酸酶非依賴性靶向基因編輯平台及其用途 HK1258288A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562192876P 2015-07-15 2015-07-15
PCT/US2016/042413 WO2017011721A1 (en) 2015-07-15 2016-07-15 Nuclease-independent targeted gene editing platform and uses thereof

Publications (1)

Publication Number Publication Date
HK1258288A1 true HK1258288A1 (zh) 2019-11-08

Family

ID=57758208

Family Applications (1)

Application Number Title Priority Date Filing Date
HK19100652.8A HK1258288A1 (zh) 2015-07-15 2019-01-15 核酸酶非依賴性靶向基因編輯平台及其用途

Country Status (9)

Country Link
US (2) US11479793B2 (zh)
EP (2) EP3322804B1 (zh)
JP (2) JP7044373B2 (zh)
CN (1) CN108291218B (zh)
CA (2) CA3168241A1 (zh)
DK (1) DK3322804T3 (zh)
ES (1) ES2892625T3 (zh)
HK (1) HK1258288A1 (zh)
WO (1) WO2017011721A1 (zh)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
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
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
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
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and their uses
CN114380922A (zh) * 2016-06-15 2022-04-22 中国科学院上海营养与健康研究所 在细胞内产生点突变的融合蛋白、其制备及用途
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3565608A4 (en) * 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENEDITATION PLATFORM INDEPENDENT OF DNA DOUBLE STRAND BREAKAGE AND USES THEREOF
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) * 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CN110446782B (zh) 2017-03-22 2024-03-01 国立大学法人神户大学 一种改变细胞所具有的dna的靶向部位的方法以及用于该方法的复合体
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN107384920B (zh) * 2017-05-10 2020-07-14 中山大学 一套基于化脓性链球菌的碱基编辑系统及其在基因编辑中的应用
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020521446A (ja) * 2017-05-25 2020-07-27 ザ ジェネラル ホスピタル コーポレイション 二部塩基エディター（ｂｂｅ）構造およびｉｉ型−ｃ−ｃａｓ９ジンクフィンガー編集
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
CN111263812A (zh) * 2017-07-31 2020-06-09 西格马-奥尔德里奇有限责任公司 用于crispr/cas激活系统的合成的指导rna
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
WO2019099943A1 (en) * 2017-11-16 2019-05-23 Astrazeneca Ab Compositions and methods for improving the efficacy of cas9-based knock-in strategies
KR102647714B1 (ko) 2018-03-19 2024-03-18 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
CN110878321B (zh) * 2018-09-06 2023-08-22 上海科技大学 一种用于肺炎克雷伯菌基因编辑的表达载体
CN109385425A (zh) * 2018-11-13 2019-02-26 中山大学 一种高特异性ABE碱基编辑系统及其在β血红蛋白病中的应用
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
CN116497067A (zh) 2019-02-13 2023-07-28 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220235348A1 (en) * 2019-05-15 2022-07-28 Board Of Regents, The University Of Texas System Crispr methods for treating cancers
JP2022533673A (ja) * 2019-05-21 2022-07-25 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いた一塩基多型編集法
EP4008784A1 (en) * 2019-08-01 2022-06-08 Astellas Pharma Inc. Guide rna for targeted-editing with functional base sequence added thereto
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
CN112481309B (zh) * 2019-09-12 2023-10-10 石家庄埃佤基因科技有限公司 Ago蛋白的用途及组合物和基因编辑方法
WO2021081264A1 (en) 2019-10-24 2021-04-29 Pairwise Plants Services, Inc. Optimized crispr-cas nucleases and base editors and methods of use thereof
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS Therapeutic editing
CN115667283A (zh) * 2020-03-03 2023-01-31 小利兰斯坦福大学董事会 Rna指导的千碱基规模基因组重组工程
BR112022017704A2 (pt) * 2020-03-04 2022-11-01 Suzhou Qi Biodesign Biotechnology Company Ltd Método e sistema de edição de genoma multiplex
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
US20230235315A1 (en) 2020-07-10 2023-07-27 Horizon Discovery Limited Method for producing genetically modified cells
CA3196996A1 (en) * 2020-11-19 2022-05-27 Baisong Lu Vectors, systems and methods for eukaryotic gene editing
US20240067954A1 (en) 2021-01-05 2024-02-29 Horizon Discovery Limited Method for producing genetically modified cells
US20240067983A1 (en) * 2021-01-05 2024-02-29 Horizon Discovery Limited Guide RNA Designs and Complexes for Tracr-less Type V Cas Systems
US20240060088A1 (en) * 2021-01-05 2024-02-22 Horizon Discovery Limited Guide RNA Designs and Complexes for Type V Cas Systems
GB202107671D0 (en) 2021-05-28 2021-07-14 Asociacion Centro De Investig Cooperativa En Nanociencias Cic Nanogune Synthetic cas proteins
EP4347808A2 (en) 2021-05-25 2024-04-10 ASOCIACIÓN CENTRO DE INVESTIGACIÓN COOPERATIVA EN NANOCIENCIAS "CIC nanoGUNE" Synthetic cas proteins
WO2024096790A1 (en) * 2022-10-31 2024-05-10 Soledits Ab Modular crispr system
CN116726198B (zh) * 2023-03-13 2024-02-13 陕西师范大学 一种抗肿瘤药物及其在脑胶质瘤治疗中的应用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US63258A (en) 1867-03-26 David king
US4625014A (en) 1984-07-10 1986-11-25 Dana-Farber Cancer Institute, Inc. Cell-delivery agent
US5061641A (en) 1988-04-01 1991-10-29 Immunomedics, Inc. Method for radiolabeling proteins
US5057301A (en) 1988-04-06 1991-10-15 Neorx Corporation Modified cellular substrates used as linkers for increased cell retention of diagnostic and therapeutic agents
WO1994012520A1 (en) 1992-11-20 1994-06-09 Enzon, Inc. Linker for linked fusion polypeptides
US6165476A (en) 1997-07-10 2000-12-26 Beth Israel Deaconess Medical Center Fusion proteins with an immunoglobulin hinge region linker
WO2008002661A2 (en) 2006-06-28 2008-01-03 The Board Of Trustees Of The Leland Stanford Junior University Fusion protein constructs
US8202983B2 (en) 2007-05-10 2012-06-19 Agilent Technologies, Inc. Thiocarbon-protecting groups for RNA synthesis
US20110104787A1 (en) 2009-11-05 2011-05-05 President And Fellows Of Harvard College Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences
SI2697257T1 (sl) 2011-04-13 2017-02-28 Bristol-Myers Squibb Company FC fuzijski proteini, ki vsebujejo nove linkerje ali razmestitve
PT2800811T (pt) 2012-05-25 2017-08-17 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
HUE035607T2 (en) 2012-06-25 2018-05-28 Brigham & Womens Hospital Inc Targeted therapeutic agents
EP3825401A1 (en) 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
CN105103045A (zh) 2012-12-18 2015-11-25 兰斯维克托公司 具有高级电场控制能力的液晶光学设备
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CN110540991B (zh) 2013-03-15 2023-10-24 通用医疗公司 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3985124A1 (en) * 2013-12-26 2022-04-20 The General Hospital Corporation Multiplex guide rnas
PL3105328T3 (pl) * 2014-02-11 2020-10-19 The Regents Of The University Of Colorado, A Body Corporate Umożliwiana przez CRISPR multipleksowa modyfikacja genomu
EP3613854A1 (en) 2014-03-05 2020-02-26 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
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
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
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and their uses
EP3565608A4 (en) 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENEDITATION PLATFORM INDEPENDENT OF DNA DOUBLE STRAND BREAKAGE AND USES THEREOF
AU2022324118A1 (en) 2021-08-06 2024-02-22 Horizon Discovery Limited Method for producing genetically modified cells

Also Published As

Publication number Publication date
EP3322804A1 (en) 2018-05-23
CN108291218B (zh) 2022-08-19
CA2992580A1 (en) 2017-01-19
WO2017011721A1 (en) 2017-01-19
US20180327784A1 (en) 2018-11-15
CA3168241A1 (en) 2017-01-19
EP3957731A1 (en) 2022-02-23
JP7364268B2 (ja) 2023-10-18
EP3322804B1 (en) 2021-09-01
CN108291218A (zh) 2018-07-17
JP2018520686A (ja) 2018-08-02
US20220267806A1 (en) 2022-08-25
US11479793B2 (en) 2022-10-25
CA2992580C (en) 2022-09-20
JP7044373B2 (ja) 2022-03-30
EP3322804A4 (en) 2019-01-30
JP2022095633A (ja) 2022-06-28
DK3322804T3 (da) 2021-10-04
ES2892625T3 (es) 2022-02-04

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