CA2902954A1 - Talen-based gene correction - Google Patents

Talen-based gene correction Download PDF

Info

Publication number
CA2902954A1
CA2902954A1 CA2902954A CA2902954A CA2902954A1 CA 2902954 A1 CA2902954 A1 CA 2902954A1 CA 2902954 A CA2902954 A CA 2902954A CA 2902954 A CA2902954 A CA 2902954A CA 2902954 A1 CA2902954 A1 CA 2902954A1
Authority
CA
Canada
Prior art keywords
cell
nucleic acid
sequence
talen
target
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.)
Abandoned
Application number
CA2902954A
Other languages
English (en)
French (fr)
Inventor
Mark J. Osborn
Jakub Tolar
Bruce Blazar
Daniel F. Voytas
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
University of Minnesota System
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 University of Minnesota System filed Critical University of Minnesota System
Publication of CA2902954A1 publication Critical patent/CA2902954A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • 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
    • C12N15/86Viral vectors
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Rheumatology (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
CA2902954A 2013-03-01 2014-02-28 Talen-based gene correction Abandoned CA2902954A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361771735P 2013-03-01 2013-03-01
US61/771,735 2013-03-01
PCT/US2014/019322 WO2014134412A1 (en) 2013-03-01 2014-02-28 Talen-based gene correction

Publications (1)

Publication Number Publication Date
CA2902954A1 true CA2902954A1 (en) 2014-09-04

Family

ID=51428832

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2902954A Abandoned CA2902954A1 (en) 2013-03-01 2014-02-28 Talen-based gene correction

Country Status (7)

Country Link
US (4) US9393257B2 (enExample)
EP (2) EP2961262B1 (enExample)
JP (3) JP6480874B2 (enExample)
AU (2) AU2014223243B2 (enExample)
CA (1) CA2902954A1 (enExample)
ES (1) ES2750550T3 (enExample)
WO (1) WO2014134412A1 (enExample)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9850497B2 (en) * 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
WO2015117021A1 (en) 2014-01-31 2015-08-06 Factor Bioscience Inc. Methods and products for nucleic acid production and delivery
US11135245B2 (en) 2014-11-17 2021-10-05 Adicet Bio, Inc. Engineered γδ T-cells
EP3543339A1 (en) 2015-02-13 2019-09-25 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN104726495B (zh) * 2015-03-25 2018-06-19 西北农林科技大学 一种基于talen介导的基因打靶敲除山羊blg的载体及重组细胞
EP4600366A3 (en) 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
US11845928B2 (en) 2015-05-04 2023-12-19 Tsinghua University Methods and kits for fragmenting DNA
KR20180029201A (ko) 2015-05-18 2018-03-20 티씨알2 테라퓨틱스 인크. 융합 단백질을 이용하는 tcr 리프로그래밍을 위한 조성물 및 방법
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
WO2017075248A1 (en) * 2015-10-27 2017-05-04 The Board Of Trustees Of The Leland Stanford Junior University Treatment of epidermolysis bullosa by injection of autologous collagen vii overexpressing leukocytes
EP3377637B1 (en) 2016-04-08 2020-03-18 Krystal Biotech, LLC Compositions for use in methods for the treatment of wounds, disorders, and diseases of the skin
JP7210286B2 (ja) 2016-05-12 2023-01-23 アディセット バイオ, インコーポレイテッド γδT細胞集団の選択的増殖方法及びその組成物
WO2018026953A1 (en) 2016-08-02 2018-02-08 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using fusion proteins
CN109803977B (zh) 2016-08-17 2023-03-17 菲克特生物科学股份有限公司 核酸产品及其施用方法
IL302917A (en) 2016-10-07 2023-07-01 Tcr2 Therapeutics Inc Compositions and methods for t-cell receptors reprogramming using fusion proteins
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
JP7291396B2 (ja) 2016-11-22 2023-06-15 ティーシーアール2 セラピューティクス インク. 融合タンパク質を用いたtcrの再プログラミングのための組成物及び方法
WO2018119298A1 (en) 2016-12-21 2018-06-28 TCR2 Therapeutics Inc. Engineered t cells for the treatment of cancer
WO2018140497A1 (en) 2017-01-25 2018-08-02 Efimov Igor R Apparatus and methods for in vitro preclinical human trials
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3585897A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders
FI3638776T3 (fi) 2017-06-14 2025-12-09 Univ Dresden Tech Menetelmiä ja välineitä genomien geneettiseen muokkaamiseen hyödyntäen suunnittelu-DNA:ta rekombinoivia entsyymejä
JP7712643B2 (ja) 2017-06-19 2025-07-24 国立大学法人東北大学 表皮水疱症の治療剤
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
IL274640B1 (en) 2017-11-15 2025-10-01 Adicet Bio Inc Methods for selective expansion of delta 3 gamma-delta T-cell populations and preparations thereof
CA3094345A1 (en) 2018-04-12 2019-10-17 Krystal Biotech, Inc. Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
WO2019204226A1 (en) 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
CN112041434A (zh) 2018-04-27 2020-12-04 克里斯托生物技术股份有限公司 用于美容性应用的编码一种或多种美容蛋白的重组核酸
EP4656196A2 (en) 2018-05-11 2025-12-03 Memorial Sloan-Kettering Cancer Center T cell receptors targeting pik3ca mutations and uses thereof
US11827892B2 (en) 2018-06-07 2023-11-28 The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization (Aro) (Volcani Center) Nucleic acid constructs and methods of using same
US20210337753A1 (en) 2018-06-07 2021-11-04 The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization Methods of regenerating and transforming cannabis
CA3109546A1 (en) * 2018-09-07 2020-03-12 Wisconsin Alumni Research Foundation Generation of hematopoietic progenitor cells from human pluripotent stem cells
JP7562515B2 (ja) 2018-09-24 2024-10-07 クリスタル バイオテック インコーポレイテッド ネザートン症候群の治療のための組成物及び方法
CN113748126A (zh) 2018-11-30 2021-12-03 因提玛生物科学公司 鉴定免疫调节基因的方法
US20220218747A1 (en) 2018-12-03 2022-07-14 Adicet Bio, Inc. Methods for selective in vivo expansion of gamma delta t-cell populations and compositions thereof
CN113454105A (zh) 2019-02-08 2021-09-28 克里斯托生物技术股份有限公司 用于递送cftr多肽的组合物和方法
AU2020253491A1 (en) 2019-04-05 2021-10-28 Rootpath Genomics, Inc. Compositions and methods for T-cell receptor gene assembly
CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
WO2020257553A1 (en) 2019-06-20 2020-12-24 Memorial Sloan-Kettering Cancer Center T cell receptors targeting pik3ca mutations and uses thereof
WO2020257684A1 (en) 2019-06-20 2020-12-24 University Of Massachusetts Compositions and methods for improved gene editing
WO2021003462A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
JP7708737B2 (ja) 2019-07-18 2025-07-15 ユニバーシティー オブ ロチェスター 細胞の細胞型選択的免疫保護
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
KR20220097875A (ko) 2019-09-03 2022-07-08 마이얼로이드 테라퓨틱스, 인크. 게놈 통합을 위한 방법 및 조성물
WO2021191870A1 (en) 2020-03-27 2021-09-30 Dcprime B.V. Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
EP4171617A1 (en) 2020-06-30 2023-05-03 Mendus B.V. Use of leukemia-derived cells in ovarian cancer vaccines
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
WO2022051418A1 (en) 2020-09-01 2022-03-10 64-X, Inc. Mammalian cells and methods for engineering the same
WO2022087380A1 (en) 2020-10-23 2022-04-28 Rootpath Genomics, Inc. Compositions and methods for t-cell receptor identification
CN116981697A (zh) 2021-01-14 2023-10-31 森迪生物科学公司 可分泌有效载荷调节
CA3203705A1 (en) 2021-01-22 2022-07-28 Erik Hans MANTING Methods of tumor vaccination
US20220305100A1 (en) 2021-03-12 2022-09-29 Dcprime B.V. Methods of vaccination and use of cd47 blockade
JP2024513826A (ja) 2021-04-02 2024-03-27 クリスタル バイオテック インコーポレイテッド がん治療のためのウイルスベクター
EP4337268A4 (en) 2021-05-11 2025-06-04 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
US11753677B2 (en) 2021-11-10 2023-09-12 Encodia, Inc. Methods for barcoding macromolecules in individual cells
EP4493681A1 (en) 2022-03-17 2025-01-22 Keshihua (Nanjing) Biotechnology Co., Ltd Synthetic tumor-infiltrating lymphocytes (tils)
WO2024039576A2 (en) 2022-08-19 2024-02-22 Memorial Sloan-Kettering Cancer Center T cell receptors targeting ras mutations and uses thereof
EP4619513A1 (en) 2022-11-18 2025-09-24 Kyoto Prefectural Public University Corporation Compositions for mitophagy induction and uses thereof
WO2025128981A1 (en) 2023-12-14 2025-06-19 Sohm, Inc. Compositions and methods for genome editing

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1533432A (zh) 2001-06-04 2004-09-29 MLʵ���ҹ������޹�˾ 高水平、大规模生产重组蛋白的组合体和方法
CA2783351C (en) 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
AU2010360293B2 (en) * 2010-09-06 2016-06-16 Temasek Life Sciences Laboratory Limited Molecular interaction between Xa10 and AvrXa10
DK2638163T3 (en) * 2010-11-12 2017-07-24 Massachusetts Gen Hospital POLYCOMB-ASSOCIATED NON-CODING RNAs
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression

Also Published As

Publication number Publication date
HK1219021A1 (en) 2017-03-24
AU2014223243A1 (en) 2015-09-10
NZ711254A (en) 2021-02-26
JP2019089821A (ja) 2019-06-13
JP6893945B2 (ja) 2021-06-23
JP2021088565A (ja) 2021-06-10
US10172880B2 (en) 2019-01-08
US20210187006A1 (en) 2021-06-24
EP2961262A1 (en) 2016-01-06
EP2961262B1 (en) 2019-07-24
EP2961262A4 (en) 2016-10-12
US20160367588A1 (en) 2016-12-22
US9393257B2 (en) 2016-07-19
JP6480874B2 (ja) 2019-03-13
AU2014223243B2 (en) 2019-10-17
WO2014134412A1 (en) 2014-09-04
ES2750550T3 (es) 2020-03-26
US10973844B2 (en) 2021-04-13
JP2016512960A (ja) 2016-05-12
US20190054111A1 (en) 2019-02-21
AU2020200307A1 (en) 2020-02-06
US20140256798A1 (en) 2014-09-11
EP3567104A1 (en) 2019-11-13

Similar Documents

Publication Publication Date Title
US10973844B2 (en) TALEN-based gene correction
ES2886194T3 (es) Endonucleasa dirigida al gen del factor VIII de coagulación sanguínea y composición para el tratamiento de la hemofilia que comprende el mismo
JP2022512703A (ja) 免疫療法のための組成物および方法
EP3468594A1 (en) Gene therapy of neuronal ceroid lipofuscinoses
US20230016422A1 (en) Engineered cells with improved protection from natural killer cell killing
WO2020131632A1 (en) Nuclease-mediated repeat expansion
JP2025081589A (ja) 改善された遺伝子編集のための組成物及び方法
US20210079394A1 (en) Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
Kim et al. Generation of TGFBI knockout ABCG2+/ABCB5+ double-positive limbal epithelial stem cells by CRISPR/Cas9-mediated genome editing
HK1219021B (en) Talen-based gene correction
NZ711254B2 (en) Talen-based gene correction
US20140377236A1 (en) Method of efficiently converting non-cardiac cells into cardiovascular cells

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20190207

EEER Examination request

Effective date: 20190207

FZDE Discontinued

Effective date: 20230829