DK3194570T3 - Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller - Google Patents

Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller Download PDF

Info

Publication number
DK3194570T3
DK3194570T3 DK15841477.1T DK15841477T DK3194570T3 DK 3194570 T3 DK3194570 T3 DK 3194570T3 DK 15841477 T DK15841477 T DK 15841477T DK 3194570 T3 DK3194570 T3 DK 3194570T3
Authority
DK
Denmark
Prior art keywords
genomification
procedures
correction
compositions
stem cells
Prior art date
Application number
DK15841477.1T
Other languages
English (en)
Inventor
Gregory J Cost
Jeffrey C Miller
Lei Zhang
Original Assignee
Sangamo Therapeutics Inc
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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Application granted granted Critical
Publication of DK3194570T3 publication Critical patent/DK3194570T3/da

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
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0645—Macrophages, e.g. Kuepfer cells in the liver; Monocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • 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
    • C12N15/87—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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
DK15841477.1T 2014-09-16 2015-09-16 Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller DK3194570T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462051159P 2014-09-16 2014-09-16
PCT/US2015/050411 WO2016044416A1 (en) 2014-09-16 2015-09-16 Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells

Publications (1)

Publication Number Publication Date
DK3194570T3 true DK3194570T3 (da) 2021-09-13

Family

ID=55533790

Family Applications (1)

Application Number Title Priority Date Filing Date
DK15841477.1T DK3194570T3 (da) 2014-09-16 2015-09-16 Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller

Country Status (18)

Country Link
US (1) US10435677B2 (da)
EP (2) EP3878948A1 (da)
JP (3) JP6722176B2 (da)
KR (1) KR20170054445A (da)
CN (2) CN113699113A (da)
AU (2) AU2015317793B2 (da)
CA (1) CA2960769A1 (da)
DK (1) DK3194570T3 (da)
ES (1) ES2886012T3 (da)
HR (1) HRP20211468T1 (da)
HU (1) HUE055583T2 (da)
LT (1) LT3194570T (da)
PL (1) PL3194570T3 (da)
PT (1) PT3194570T (da)
RS (1) RS62334B1 (da)
SG (2) SG10202011572XA (da)
SI (1) SI3194570T1 (da)
WO (1) WO2016044416A1 (da)

Families Citing this family (66)

* 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
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
CA2986310A1 (en) * 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
AU2016261927B2 (en) * 2015-05-12 2022-04-07 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US20200208111A1 (en) * 2016-06-09 2020-07-02 Branden S. Moriarity Genome-edited nk cell and methods of making and using
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CA3047542A1 (en) * 2016-12-19 2018-06-28 The Uab Research Foundation Mass spectrometric standards for hemoglobin beta and hemoglobin beta sickle and uses thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
BR112019015355A2 (pt) * 2017-01-26 2020-05-19 Sangamo Therapeutics Inc modificação genética de células b
EP3574088A4 (en) 2017-01-27 2020-09-23 Children's Hospital Medical Center PROCESSES FOR IMPROVING HEMATOPOIETIC STEM CELL TRANSPLANT ACTIVITY
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
RU2019130504A (ru) 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11655275B2 (en) 2017-05-03 2023-05-23 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
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
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
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
US20190314407A1 (en) * 2017-10-31 2019-10-17 Magenta Therapeutics Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells
BR112020008568A2 (pt) * 2017-11-09 2020-10-06 Sangamo Therapeutics, Inc. modificação genética de gene de proteína contendo sh2 indutível por citoquina (cish)
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3511412A1 (en) * 2018-01-12 2019-07-17 Genethon Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3575402A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
KR20210058816A (ko) 2018-08-18 2021-05-24 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 원 위치에 유전자 편집
US20210340501A1 (en) * 2018-08-27 2021-11-04 The Regents Of The University Of California Methods to Control Viral Infection in Mammalian Cells
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
GB201815670D0 (en) * 2018-09-26 2018-11-07 Univ Oxford Innovation Ltd Protein editing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020179931A1 (ja) * 2019-03-07 2020-09-10 国立大学法人 東京医科歯科大学 ハイスループット遺伝子編集技術
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CA3132167A1 (en) 2019-04-02 2020-10-08 Weston P. MILLER IV Methods for the treatment of beta-thalassemia
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3137568A1 (en) * 2019-04-25 2020-10-29 Actinium Pharmaceuticals, Inc. Compositions and methods of immunodepletion for the treatment of malignant and non-malignant hematological diseases
EP3770264A1 (en) * 2019-07-22 2021-01-27 Genethon Precise integration using nuclease targeted idlv
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20240124561A1 (en) * 2019-10-17 2024-04-18 Minicircle Inc. Nucleic acids encoding hiv neutralizing antibodies and uses thereof
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
WO2022094363A1 (en) * 2020-11-01 2022-05-05 University of South Alabama Foundation for Research and Commercialization Barcoded cells engineered with heterozygous genetic diversity
CN115232811B (zh) * 2021-06-28 2025-10-28 南京启真基因工程有限公司 用于构建hbb基因突变的镰刀型细胞贫血症模型猪核移植供体细胞的方法及应用
CA3225981A1 (en) * 2021-07-01 2023-01-05 Bluebird Bio, Inc. Methods
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194124A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids

Family Cites Families (108)

* 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
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
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
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
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
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
JP4285766B2 (ja) 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
JP4540754B2 (ja) * 1996-06-04 2010-09-08 ユニバーシティ オブ ユタ リサーチ ファウンデーション Ｐｃｒ中のハイブリダイゼーションのモニタリング
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US7030846B2 (en) 2001-07-10 2006-04-18 Samsung Electronics Co., Ltd. Color correction liquid crystal display and method of driving same
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
WO2003027247A2 (en) 2001-09-24 2003-04-03 Sangamo Biosciences, Inc. Modulation of stem cells using zinc finger proteins
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
AU2003261970A1 (en) * 2002-09-06 2004-03-29 Wakunaga Pharmaceutical Co., Ltd. Method of identifying nucleic acid having polymorphism sequence site
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
JP5139271B2 (ja) 2005-05-24 2013-02-06 ホワイトヘッド インスティチュート フォー バイオメディカル リサーチ 培養した造血幹細胞を拡大しかつ分析する方法
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
RU2425876C2 (ru) 2006-03-24 2011-08-10 Чилдрен'З Медикал Сентер Корпорейшн Способ модулирования роста гематопоэтических стволовых клеток
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
DK3339321T3 (da) 2008-08-28 2021-06-21 Taiga Biotechnologies Inc Modulatorer af myc, fremgangsmåder til anvendelse af samme og fremgangsmåder til at identificere stoffer der modulerer myc
EP2352369B1 (en) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genome editing in rats using zinc-finger nucleases
CN102333868B (zh) 2008-12-17 2015-01-07 陶氏益农公司 靶向整合入Zp15 基因座
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
MX336846B (es) * 2010-01-22 2016-02-03 Sangamo Biosciences Inc Alteracion genomica dirigida.
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
WO2012071513A2 (en) 2010-11-24 2012-05-31 Hong Gao Expanding hematopoietic stem cells
WO2013019745A1 (en) * 2011-07-29 2013-02-07 Georgia Health Sciences University Methods and compositions for genetically modifiying cells
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
AU2013259647B2 (en) * 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
US11120889B2 (en) * 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
JP6329537B2 (ja) * 2012-07-11 2018-05-23 サンガモ セラピューティクス， インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
US9789135B2 (en) 2012-07-20 2017-10-17 Taiga Biotechnologies, Inc. Enhanced reconstitution and autoreconstitution of the hematopoietic compartment
SG10201701601WA (en) * 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease

Also Published As

Publication number Publication date
PL3194570T3 (pl) 2021-12-20
US20180187173A1 (en) 2018-07-05
AU2022200835A1 (en) 2022-03-03
EP3194570A4 (en) 2018-04-04
CA2960769A1 (en) 2016-03-24
WO2016044416A1 (en) 2016-03-24
ES2886012T3 (es) 2021-12-16
EP3194570A1 (en) 2017-07-26
EP3878948A1 (en) 2021-09-15
CN113699113A (zh) 2021-11-26
PT3194570T (pt) 2021-08-06
CN106795488A (zh) 2017-05-31
JP2019193679A (ja) 2019-11-07
HRP20211468T1 (hr) 2021-12-24
KR20170054445A (ko) 2017-05-17
RS62334B1 (sr) 2021-10-29
HUE055583T2 (hu) 2021-12-28
LT3194570T (lt) 2021-09-27
AU2015317793B2 (en) 2021-11-11
SG11201702134RA (en) 2017-04-27
JP6722176B2 (ja) 2020-07-15
JP2017532955A (ja) 2017-11-09
AU2015317793A1 (en) 2017-03-30
SG10202011572XA (en) 2021-01-28
SI3194570T1 (sl) 2022-01-31
JP2021182931A (ja) 2021-12-02
US10435677B2 (en) 2019-10-08
JP6929585B2 (ja) 2021-09-01
EP3194570B1 (en) 2021-06-30
CN106795488B (zh) 2021-03-30

Similar Documents

Publication Publication Date Title
DK3194570T3 (da) Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller
GB2535253B (en) Compositions and methods
DK3102673T3 (da) Fremgangsmåder og sammensætninger til behandling af beta-talassæmi
DK3387134T3 (da) Fremgangsmåder og sammensætninger til øget nukleasemedieret genommodifikation og reducerede virkninger uden for målstedet
DK3268471T3 (da) Sammensætninger og fremgangsmåder omfattende lg12-clade-proteasevarianter
GB201609438D0 (en) Compositions and methods for cell targeted HPV treatment
DK3234133T3 (da) Crispr-baserede sammensætninger og fremgangsmåder til anvendelse
DK3125927T3 (da) Fremgangsmåder og sammensætninger til immunmodulering
DK3757206T3 (da) Transduktionsfremgangsmåde og cellebehandling
DK3102189T3 (da) Sammensætning og fremgangsmåde til at fremme søvn
SG11201708524VA (en) Self-foaming compositions and methods
DK3018183T3 (da) Halogenolefinbaseret sammensætning og anvendelse deraf
DK3218358T3 (da) Ifluorometyl-aminopyridiner og difluorometyl-aminopyrimidiner
DK3134130T3 (da) Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier
DK3102232T3 (da) Fremgangsmåder og sammensætninger til behandling af abnorm cellevækst
DK3368663T3 (da) Virusfri cellelinjer og fremgangsmåder til opnåelse deraf
DK3586821T3 (da) Stamcellestimulerende sammensætninger og metoder
IL246518A0 (en) The compositions, including nanosheets, and methods to prevent friction wear and lubrication
DK3220750T3 (da) Oral rehydreringssammensætning og fremgangsmåder dertil
DK3240892T3 (da) Rpe-cellepopulationer og fremgangsmåder til dannelse deraf
DK3110446T3 (da) Fremgangsmåder og sammensætninger til behandling af Siglec-8-associerede sygdomme
DK3148517T3 (da) Sammensætninger og fremgangsmåder til fremme af knogledannelse
DK3212776T3 (da) Sammensætninger og fremgangsmåder i forbindelse med beta-glucosidase
DK3389620T3 (da) Stamcellestimulerende sammensætninger og fremgangsmåder til behandling af melasma
DK3104960T3 (da) Reaktiv sammensætning baseret på natriumbicarbonat og fremgangsmåde til fremstilling deraf