JP2015523860A - DNAの修復、変更および置き換えのための道具としてのスーパーコイルMiniVector - Google Patents

DNAの修復、変更および置き換えのための道具としてのスーパーコイルMiniVector Download PDF

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JP2015523860A
JP2015523860A JP2015515201A JP2015515201A JP2015523860A JP 2015523860 A JP2015523860 A JP 2015523860A JP 2015515201 A JP2015515201 A JP 2015515201A JP 2015515201 A JP2015515201 A JP 2015515201A JP 2015523860 A JP2015523860 A JP 2015523860A
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minivector
nucleic acid
template
dna sequence
site
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Japanese (ja)
Inventor
ゼチードリッチ,イー・リン
フォグ,ジョナサン
カタニーズ,ジュニア,ダニエル・ジェームズ
マイゼル,ナンシー
ハンバート,オリヴィエ
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Baylor College of Medicine
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Baylor College of Medicine
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    • 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
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/8201Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2800/24Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2015515201A 2012-05-30 2013-05-30 DNAの修復、変更および置き換えのための道具としてのスーパーコイルMiniVector Ceased JP2015523860A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261653279P 2012-05-30 2012-05-30
US61/653,279 2012-05-30
PCT/US2013/043433 WO2013181440A1 (en) 2012-05-30 2013-05-30 Supercoiled minivectors as a tool for dna repair, alteration and replacement

Publications (1)

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JP2015523860A true JP2015523860A (ja) 2015-08-20

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US (2) US20140056868A1 (enExample)
EP (1) EP2854866A4 (enExample)
JP (1) JP2015523860A (enExample)
KR (1) KR20150027756A (enExample)
AU (1) AU2013267350A1 (enExample)
BR (1) BR112014030007A2 (enExample)
CA (1) CA2876860A1 (enExample)
IN (1) IN2014DN10996A (enExample)
MX (1) MX2014014650A (enExample)
WO (1) WO2013181440A1 (enExample)

Families Citing this family (77)

* 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
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
NZ714353A (en) 2012-05-25 2017-05-26 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
CA2905432C (en) 2013-03-14 2021-09-14 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CN105637087A (zh) 2013-09-18 2016-06-01 科马布有限公司 方法、细胞与生物体
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US11318206B2 (en) 2014-03-28 2022-05-03 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules
KR20160138210A (ko) * 2014-03-28 2016-12-02 아포센스 엘티디. 분자의 막횡단 전달을 위한 화합물 및 방법
JP2017515464A (ja) 2014-04-10 2017-06-15 シアトル チルドレンズ ホスピタル, ディービーエー シアトル チルドレンズ リサーチ インスティテュート 細胞免疫療法のための方法および組成物
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US11071289B2 (en) 2014-08-14 2021-07-27 Biocytogen Boston Corp DNA knock-in system
US20180250424A1 (en) 2014-10-10 2018-09-06 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
CN105821072A (zh) * 2015-01-23 2016-08-03 深圳华大基因研究院 用于DNA组装的CRISPR-Cas9系统及DNA组装方法
AU2016306209B2 (en) 2015-08-07 2023-07-06 Seattle Children's Hospital (dba Seattle Children's Research Institute) Bispecific CAR T-cells for solid tumor targeting
EP3786294A1 (en) 2015-09-24 2021-03-03 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
WO2017147278A1 (en) 2016-02-25 2017-08-31 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology
EP3219803A1 (en) * 2016-03-15 2017-09-20 Max-Delbrück-Centrum für Molekulare Medizin Enhanced sleeping beauty transposons, kits and methods of transposition
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2017207589A1 (en) 2016-06-01 2017-12-07 Kws Saat Se Hybrid nucleic acid sequences for genome engineering
CN109477130B (zh) 2016-07-01 2022-08-30 微软技术许可有限责任公司 通过迭代dna编辑的存储
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
US10892034B2 (en) 2016-07-01 2021-01-12 Microsoft Technology Licensing, Llc Use of homology direct repair to record timing of a molecular event
CA3032699A1 (en) 2016-08-03 2018-02-08 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
EP3523319B1 (en) 2016-10-10 2023-06-28 Limagrain Europe Nucleic acid encoding sm1 resistance to orange wheat blossom midge and method of use
US11306324B2 (en) 2016-10-14 2022-04-19 President And Fellows Of Harvard College AAV delivery of nucleobase editors
CN109906030B (zh) 2016-11-04 2022-03-18 安健基因公司 用于产生仅重链抗体的经基因修饰的非人动物和方法
EP3551197B1 (en) 2016-12-12 2023-11-22 Seattle Children's Hospital (DBA Seattle Children's Research Institute) Chimeric transcription factor variants with augmented sensitivity to drug ligand induction of transgene expression in mammalian cells
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2018205898B2 (en) 2017-01-09 2020-12-10 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
US20180305701A1 (en) 2017-03-14 2018-10-25 Baylor College Of Medicine Targeting minivectors to specific tissue using shape
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
WO2019068800A1 (en) 2017-10-05 2019-04-11 Biogemma IMPROVED YIELD IN PLANTS BY OVEREXPRESSION OF A TRHEHALOSE-6 PHOSPHATE SYNTHASE
JP2021500036A (ja) 2017-10-16 2021-01-07 ザ ブロード インスティテュート, インコーポレーテッドThe Broad Institute, Inc. アデノシン塩基編集因子の使用
AU2018357926B2 (en) 2017-10-31 2023-04-06 Limagrain Europe Wheat comprising male fertility restorer alleles
US20200048716A1 (en) 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
BR112020008919A2 (pt) * 2017-11-21 2020-10-20 Monsanto Technology Llc plantas modificadas com traços aprimorados
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN111615404A (zh) 2018-01-01 2020-09-01 艾博森斯有限公司 用于分子的跨膜递送的化合物和方法
GB201805865D0 (en) 2018-04-09 2018-05-23 Innes John Centre Genes
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020161261A1 (en) 2019-02-06 2020-08-13 Vilmorin & Cie New gene responsible for cytoplasmic male sterility
KR20210143230A (ko) 2019-03-19 2021-11-26 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020309724A1 (en) 2019-07-05 2022-02-24 Centre National De La Recherche Scientifique Method for increasing yield in plants
FR3099178A1 (fr) 2019-07-24 2021-01-29 Soltis Tournesol à teneur élevée en acide oléique et procédé d’obtention
TW202124722A (zh) 2019-09-18 2021-07-01 美商英特佳樂帝克醫療公司 合成dna載體及其使用方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN121109454A (zh) 2019-11-06 2025-12-12 青岛清原化合物有限公司 在生物体内创制新基因的方法及应用
WO2021202832A1 (en) * 2020-04-03 2021-10-07 Progenitor Life Sciences Targeting tapasin and tap complex to improve cellular immune-compatibility
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2021247507A1 (en) 2020-06-01 2021-12-09 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
WO2022240806A1 (en) 2021-05-11 2022-11-17 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
US20240100189A1 (en) 2021-09-10 2024-03-28 Twister Biotech, Inc Using minivectors to treat idiopathic pulmonary fibrosis
US20240093226A1 (en) 2021-09-10 2024-03-21 Baylor College Of Medicine Ultrapure minivectors for gene therapy
US20230190955A1 (en) 2021-10-21 2023-06-22 Baylor College Of Medicine Treatment of liver cancer or liver fibrosis

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU1100201A (en) * 1999-10-28 2001-05-08 Board Of Trustees Of The Leland Stanford Junior University Methods of in vivo gene transfer using a sleeping beauty transposon system
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US8460924B2 (en) * 2009-10-16 2013-06-11 Baylor College Of Medicine Supercoiled minivectors™ for gene therapy applications
US9125385B2 (en) * 2010-11-12 2015-09-08 The Board Of Trustees Of The Leland Stanford Junior University Site-directed integration of transgenes in mammals
DK2839013T3 (da) * 2012-04-18 2020-09-14 Univ Leland Stanford Junior Ikke-disruptiv-gen-targetering

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CA2876860A1 (en) 2013-12-05
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KR20150027756A (ko) 2015-03-12
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