AU2017295898B2 - Methods and compositions for genome editing in non-dividing cells - Google Patents

Methods and compositions for genome editing in non-dividing cells Download PDF

Info

Publication number
AU2017295898B2
AU2017295898B2 AU2017295898A AU2017295898A AU2017295898B2 AU 2017295898 B2 AU2017295898 B2 AU 2017295898B2 AU 2017295898 A AU2017295898 A AU 2017295898A AU 2017295898 A AU2017295898 A AU 2017295898A AU 2017295898 B2 AU2017295898 B2 AU 2017295898B2
Authority
AU
Australia
Prior art keywords
sequence
nuclease
vector
cell
genome
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.)
Active
Application number
AU2017295898A
Other languages
English (en)
Other versions
AU2017295898A1 (en
Inventor
Reyna HERNANDEZ-BENITEZ
Juan Carlos Izpisua Belmonte
Keiichiro Suzuki
Yuji TSUNEKAWA
Jun Wu
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.)
Salk Institute for Biological Studies
Original Assignee
Salk Institute for Biological Studies
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 Salk Institute for Biological Studies filed Critical Salk Institute for Biological Studies
Publication of AU2017295898A1 publication Critical patent/AU2017295898A1/en
Application granted granted Critical
Publication of AU2017295898B2 publication Critical patent/AU2017295898B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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 [CRISPR]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2017295898A 2016-07-15 2017-07-14 Methods and compositions for genome editing in non-dividing cells Active AU2017295898B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662363164P 2016-07-15 2016-07-15
US62/363,164 2016-07-15
PCT/US2017/042151 WO2018013932A1 (en) 2016-07-15 2017-07-14 Methods and compositions for genome editing in non-dividing cells

Publications (2)

Publication Number Publication Date
AU2017295898A1 AU2017295898A1 (en) 2019-02-21
AU2017295898B2 true AU2017295898B2 (en) 2023-08-17

Family

ID=59564227

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017295898A Active AU2017295898B2 (en) 2016-07-15 2017-07-14 Methods and compositions for genome editing in non-dividing cells

Country Status (6)

Country Link
US (3) US11674158B2 (OSRAM)
EP (2) EP3485023B1 (OSRAM)
JP (3) JP2019524098A (OSRAM)
AU (1) AU2017295898B2 (OSRAM)
CA (1) CA3030587A1 (OSRAM)
WO (1) WO2018013932A1 (OSRAM)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2017053879A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
AU2017295898B2 (en) 2016-07-15 2023-08-17 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 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
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
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
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
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)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
JP2020534812A (ja) * 2017-09-08 2020-12-03 ライフ テクノロジーズ コーポレイション 改良された相同組換えおよびその組成物のための方法
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
JP7498499B2 (ja) * 2018-05-03 2024-06-12 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 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019239361A1 (en) * 2018-06-14 2019-12-19 Novartis Ag Method for sequence insertion using crispr
AU2019326392A1 (en) 2018-08-18 2021-03-11 Dana-Farber Cancer Institute, Inc. In situ gene editing
AU2019326617A1 (en) * 2018-08-24 2021-03-18 Locanabio, Inc. FASL immunomodulatory gene therapy compositions and methods for use
WO2020051360A1 (en) * 2018-09-05 2020-03-12 The Broad Institute, Inc. Base editing for treating hutchinson-gilford progeria syndrome
CA3116452A1 (en) * 2018-10-15 2020-04-23 Fondazione Telethon Genome editing methods and constructs
MX2021004277A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para expresar el factor ix.
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
AU2019369527A1 (en) * 2018-10-30 2021-05-20 Bayer Healthcare Llc Compositions and methods for NHEJ-mediated genome editing
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3924470A4 (en) * 2019-02-15 2022-12-14 Exhaura, Ltd. DUAL LEUCINE ZIPPER KINASE INHIBITORS FOR GENE THERAPY
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
JP2022529424A (ja) * 2019-04-14 2022-06-22 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにしたゲノム編集組成物
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
KR20220034833A (ko) 2019-07-12 2022-03-18 리켄 현성형 변이 유전자에 유래하는 질환의 치료제
WO2021034336A1 (en) * 2019-08-22 2021-02-25 Salk Institute For Biological Studies Compositions and methods for in vivo gene editing
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
CN112741906B (zh) * 2019-10-31 2022-07-05 华东师范大学 一种用于治疗b型血友病的产品
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.
JP2023541444A (ja) * 2020-09-15 2023-10-02 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィー患者における多様なｄｍｄ変異の補正のためのａａｖ媒介性の相同性非依存的標的化組み込み遺伝子編集
CA3218195A1 (en) * 2021-05-07 2022-11-10 Robin Ali Abca4 genome editing
JP2024518793A (ja) * 2021-05-27 2024-05-02 アストラゼネカ・アクチエボラーグ 向上した安定性を有するｃａｓ９エフェクタータンパク質
KR20240126449A (ko) 2021-11-19 2024-08-20 더 유니버시티 오브 브리스톨 황반변성 치료를 위한 재료 및 방법
JP2025515030A (ja) * 2022-05-02 2025-05-13 フォンダジオン テレソン イーティーエス 遺伝子編集用の相同性非依存性標的組込み
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
CA2163129A1 (en) 1993-05-17 1994-11-24 Flossie Wong-Staal Ribozyme gene therapy for hiv infection and aids
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
JP2010539931A (ja) 2007-09-27 2010-12-24 サンガモ バイオサイエンシーズ， インコーポレイテッド ジンクフィンガーヌクレアーゼを使用したゼブラフィッシュにおけるゲノム編集
ES2696825T3 (es) 2009-12-10 2019-01-18 Univ Minnesota Modificación del ADN inducida por el efector TAL
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9752115B2 (en) 2014-02-26 2017-09-05 Maine Medical Center Research Institute Culture conditions for expansion of nephron progenitor cells
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
MX391205B (es) 2015-09-11 2025-03-11 Astellas Pharma Inc Metodo para producir celulas progenitoras renales.
US20170205396A1 (en) 2016-01-15 2017-07-20 Salk Institute For Biological Studies Systems and methods for culturing nephron progenitor cells
US20170283777A1 (en) 2016-03-25 2017-10-05 Salk Institute For Biological Studies Mammalian chimeric complementation
AU2017295898B2 (en) 2016-07-15 2023-08-17 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
M. MARESCA, LIN V. G., GUO N., YANG Y.: "Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-designed nuclease-mediated targeted integration through nonhomologous end joining", GENOME RESEARCH, COLD SPRING HARBOR LABORATORY PRESS, vol. 23, no. 3, 1 March 2013 (2013-03-01), pages 539 - 546, XP055077484, ISSN: 10889051, DOI: 10.1101/gr.145441.112 *
S. J. ORLANDO, SANTIAGO Y., DEKELVER R. C., FREYVERT Y., BOYDSTON E. A., MOEHLE E. A., CHOI V. M., GOPALAN S. M., LOU J. F., LI J.: "Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 38, no. 15, 1 August 2010 (2010-08-01), GB , pages e152 - e152 - 15, XP055076783, ISSN: 0305-1048, DOI: 10.1093/nar/gkq512 *
SANDRA CRISTEA, FREYVERT, SANTIAGO, HOLES, URNOV, GREGORY, COST: "In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration", BIOTECHNOLOGY AND BIOENGINEERING, vol. 110, no. 3, 1 March 2013 (2013-03-01), pages 871 - 880, XP055076901, DOI: 10.1002/bit.24733 *
SHOTA NAKADE, TAKUYA TSUBOTA, YUTO SAKANE, SATOSHI KUME, NAOAKI SAKAMOTO, MASANOBU OBARA, TAKAAKI DAIMON, HIDEKI SEZUTSU, TAKASHI : "Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9", NATURE COMMUNICATIONS, vol. 5, pages 5560, XP055342780, DOI: 10.1038/ncomms6560 *
T. O. AUER, K. DUROURE, A. DE CIAN, J.-P. CONCORDET, F. DEL BENE: "Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair", GENOME RESEARCH, COLD SPRING HARBOR LABORATORY PRESS, US, vol. 24, no. 1, 1 January 2014 (2014-01-01), US , pages 142 - 153, XP055237091, ISSN: 1088-9051, DOI: 10.1101/gr.161638.113 *
TETSUSHI SAKUMA, SHOTA NAKADE, YUTO SAKANE, KEN-ICHI T SUZUKI, TAKASHI YAMAMOTO: "MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems", NATURE PROTOCOLS, NATURE PUBLISHING GROUP, GB, vol. 11, no. 1, GB , pages 118 - 133, XP055388413, ISSN: 1754-2189, DOI: 10.1038/nprot.2015.140 *
XIANGJUN HE, TAN CHUNLAI, WANG FENG, WANG YAOFENG, ZHOU RUI, CUI DEXUAN, YOU WENXING, ZHAO HUI, REN JIANWEI, FENG BO: "Knock-in of large reporter genes in human cells via CRISPR/Cas9-induced homology-dependent and independent DNA repair", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 44, no. 9, 19 May 2016 (2016-05-19), GB , pages e85 - e85, XP055415869, ISSN: 0305-1048, DOI: 10.1093/nar/gkw064 *

Also Published As

Publication number Publication date
US11959094B2 (en) 2024-04-16
US20190225991A1 (en) 2019-07-25
EP3485023C0 (en) 2023-11-15
US20230399663A1 (en) 2023-12-14
JP2024119833A (ja) 2024-09-03
AU2017295898A1 (en) 2019-02-21
EP4321623A3 (en) 2024-05-15
JP7493563B2 (ja) 2024-05-31
EP3485023B1 (en) 2023-11-15
JP2023092453A (ja) 2023-07-03
WO2018013932A1 (en) 2018-01-18
US11674158B2 (en) 2023-06-13
US20240409961A1 (en) 2024-12-12
CA3030587A1 (en) 2018-01-18
EP4321623A2 (en) 2024-02-14
EP3485023A1 (en) 2019-05-22
JP2019524098A (ja) 2019-09-05

Similar Documents

Publication Publication Date Title
US11959094B2 (en) Methods and compositions for genome editing in non-dividing cells
JP6506185B2 (ja) 幹細胞への標的組込み
EP3289080B1 (en) Gene therapy for autosomal dominant diseases
US20250222140A1 (en) Genome editing methods and constructs
US20160046683A1 (en) Methods and compositions for gene correction
JP7628319B2 (ja) ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法
CN113423831B (zh) 核酸酶介导的重复扩增
US20210230606A1 (en) Methods and compositions for enhancing functional myelin production
JP7756639B2 (ja) Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略
CN110997924A (zh) 用于在肝中表达感兴趣的蛋白的平台
HK40102358A (en) Methods and compositions for genome editing in non-dividing cells
HK40075787A (en) Gene therapy for autosomal dominant diseases
HK1251615B (en) Gene therapy for autosomal dominant diseases

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)