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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Cell Biology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (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 (enExample)
EP (2) EP3485023B1 (enExample)
JP (3) JP2019524098A (enExample)
AU (1) AU2017295898B2 (enExample)
CA (1) CA3030587A1 (enExample)
WO (1) WO2018013932A1 (enExample)

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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3204496A1 (en) 2014-10-10 2017-08-16 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
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
CA2999500A1 (en) 2015-09-24 2017-03-30 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
MX2018005377A (es) 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11674158B2 (en) 2016-07-15 2023-06-13 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CN111315889A (zh) * 2017-09-08 2020-06-19 生命技术公司 提高同源重组的方法和其组合物
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20210363546A1 (en) * 2018-05-03 2021-11-25 President And Fellows Of Harvard College In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells
WO2019226953A1 (en) 2018-05-23 2019-11-28 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
EP3836974A4 (en) 2018-08-18 2022-06-15 President and Fellows of Harvard College IN SITU GENE EDIT
US20220175960A1 (en) * 2018-08-24 2022-06-09 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
US12285495B2 (en) 2018-10-15 2025-04-29 Fondazione Telethon Ets Genome editing methods and constructs
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US20220000933A1 (en) * 2018-10-30 2022-01-06 Crispr Therapeutics Ag Compositions and methods for nhej-mediated genome editing
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020168111A1 (en) * 2019-02-15 2020-08-20 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
EP3930766A4 (en) * 2019-04-14 2023-02-22 Duke University CRISPR/CAS-BASED GENE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
US20220249698A1 (en) * 2019-07-12 2022-08-11 Riken Therapeutic agent for disease caused by dominant mutant gene
CA3152056A1 (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
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
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
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
AU2021345112A1 (en) * 2020-09-15 2023-04-27 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
US20240425877A1 (en) * 2021-05-07 2024-12-26 Ucl Business Ltd Abca4 genome editing
WO2022248645A1 (en) * 2021-05-27 2022-12-01 Astrazeneca Ab Cas9 effector proteins with enhanced stability
CA3238451A1 (en) 2021-11-19 2023-05-25 Ying Kai CHAN Materials and methods for treatment of macular degeneration
IL316555A (en) * 2022-05-02 2024-12-01 Fond Telethon Ets Homology-independent directed integration for gene editing
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
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
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
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
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
AU680921B2 (en) 1993-05-17 1997-08-14 Regents Of The University Of California, The Ribozyme gene therapy for HIV infection and AIDS
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
WO1999045132A1 (en) 1998-03-02 1999-09-10 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
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
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
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
CA2700170A1 (en) 2007-09-27 2009-04-02 Sangamo Biosciences, Inc. Genomic editing in zebrafish using zinc finger nucleases
CA2783351C (en) 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
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
JP6830893B2 (ja) 2015-09-11 2021-02-17 アステラス製薬株式会社 腎前駆細胞を製造する方法
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
US11674158B2 (en) 2016-07-15 2023-06-13 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
US20230399663A1 (en) 2023-12-14
US20240409961A1 (en) 2024-12-12
JP2019524098A (ja) 2019-09-05
EP3485023B1 (en) 2023-11-15
AU2017295898A1 (en) 2019-02-21
JP2023092453A (ja) 2023-07-03
US20190225991A1 (en) 2019-07-25
EP4321623A2 (en) 2024-02-14
EP3485023C0 (en) 2023-11-15
US11674158B2 (en) 2023-06-13
JP2024119833A (ja) 2024-09-03
EP3485023A1 (en) 2019-05-22
JP7493563B2 (ja) 2024-05-31
EP4321623A3 (en) 2024-05-15
WO2018013932A1 (en) 2018-01-18
CA3030587A1 (en) 2018-01-18

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
CN113423831B (zh) 核酸酶介导的重复扩增
JP7628319B2 (ja) ｉｎ ｖｉｖｏ遺伝子編集のための組成物および方法
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)