CN116113696A - 基因编辑以改善关节功能 - Google Patents

基因编辑以改善关节功能 Download PDF

Info

Publication number
CN116113696A
CN116113696A CN202180054064.8A CN202180054064A CN116113696A CN 116113696 A CN116113696 A CN 116113696A CN 202180054064 A CN202180054064 A CN 202180054064A CN 116113696 A CN116113696 A CN 116113696A
Authority
CN
China
Prior art keywords
gene
exon
guide rna
sequence
seq
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.)
Pending
Application number
CN202180054064.8A
Other languages
English (en)
Chinese (zh)
Inventor
彼得·J·米利特
伊恩·阿拉斯代尔·拉塞尔
马修·J·艾伦
乔治·根茨
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.)
Ozer Biotherapy Co
Original Assignee
Ozer Biotherapy Co
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 Ozer Biotherapy Co filed Critical Ozer Biotherapy Co
Publication of CN116113696A publication Critical patent/CN116113696A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
CN202180054064.8A 2020-07-16 2021-07-16 基因编辑以改善关节功能 Pending CN116113696A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063052881P 2020-07-16 2020-07-16
US63/052,881 2020-07-16
US202063055808P 2020-07-23 2020-07-23
US63/055,808 2020-07-23
PCT/US2021/042100 WO2022016121A2 (en) 2020-07-16 2021-07-16 Gene editing to improve joint function

Publications (1)

Publication Number Publication Date
CN116113696A true CN116113696A (zh) 2023-05-12

Family

ID=79555038

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180054064.8A Pending CN116113696A (zh) 2020-07-16 2021-07-16 基因编辑以改善关节功能

Country Status (11)

Country Link
US (1) US20230257779A1 (https=)
EP (1) EP4182461A4 (https=)
JP (1) JP2023535351A (https=)
KR (1) KR20230041729A (https=)
CN (1) CN116113696A (https=)
AU (1) AU2021308079A1 (https=)
BR (1) BR112023000738A2 (https=)
CA (1) CA3186119A1 (https=)
IL (1) IL299846A (https=)
MX (1) MX2023000671A (https=)
WO (2) WO2022016100A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4694913A2 (en) * 2023-04-11 2026-02-18 Orthobio Therapeutics, Inc. Lipid nanoparticles for gene editing systems
WO2025024814A2 (en) * 2023-07-26 2025-01-30 Orthobio Therapeutics, Inc. Transmembrane receptor gene editing
CN117448379A (zh) * 2023-12-22 2024-01-26 上海元戊医学技术有限公司 iPSC来源的IL-10蛋白过表达MSC细胞株的构建方法及其应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9711040D0 (en) * 1997-05-29 1997-07-23 Duff Gordon W Prediction of inflammatory disease
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
US20160279202A1 (en) * 2013-10-31 2016-09-29 Ramot At Tel-Aviv University Ltd. Interleukin-1 (il-1) inhibitors for treating fertility disorders
US20170240901A1 (en) * 2014-02-26 2017-08-24 The University Of Liverpool Nucleic acids, and uses therof
US20170335331A1 (en) * 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
WO2017223107A1 (en) * 2016-06-20 2017-12-28 Unity Biotechnology, Inc. Genome modifying enzyme therapy for diseases modulated by senescent cells
WO2018031950A1 (en) * 2016-08-12 2018-02-15 Caribou Biosciences, Inc. Protein engineering methods

Also Published As

Publication number Publication date
IL299846A (en) 2023-03-01
WO2022016100A2 (en) 2022-01-20
JP2023535351A (ja) 2023-08-17
US20230257779A1 (en) 2023-08-17
WO2022016100A3 (en) 2022-03-17
MX2023000671A (es) 2023-05-16
BR112023000738A2 (pt) 2023-03-21
AU2021308079A8 (en) 2023-03-16
WO2022016121A3 (en) 2022-03-10
EP4182461A4 (en) 2025-03-19
KR20230041729A (ko) 2023-03-24
WO2022016121A2 (en) 2022-01-20
AU2021308079A1 (en) 2023-03-02
EP4182461A2 (en) 2023-05-24
CA3186119A1 (en) 2022-01-20

Similar Documents

Publication Publication Date Title
US11773409B2 (en) CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
ES2344660T3 (es) Uso de virus adeno-asociados recombinantes en la preparacion de un medicamento para la terapia genica mediante celulas musculares.
CN116113696A (zh) 基因编辑以改善关节功能
US20240299579A1 (en) Gene editing to improve joint function
US12419968B2 (en) Gene editing to improve joint function
US20230235321A1 (en) Gene editing to improve joint function
HK40120118A (zh) 用於改善关节功能的基因编辑
CN112601454A (zh) 用于治疗杜兴肌营养不良的组合物和方法
Odabas et al. Exon7 targeted CRISPR-prime editing approaches for SMN2 gene editing in spinal muscular atrophy (SMA) disease can increase in vitro SMN expression
Chesnokova et al. Regulatory Elements for Gene Therapy of Epilepsy
CN116323941A (zh) 通过诱导抗肌萎缩蛋白相关蛋白调节元件内的突变增强细胞中抗肌萎缩蛋白相关蛋白表达及其治疗用途
WO2021168216A1 (en) Crispr/cas9 correction of mutations in dystrophin exons 43, 45 and 52
US20240175007A1 (en) Compositions for and methods of improving directed evolution of biomolecules
HK40063149A (en) Gene editing to improve joint function
US20200113972A1 (en) Vegf gene therapy for tendon and ligament injuries
WO2026006334A1 (en) Dux4 vectors and engineered guide rnas
EP4713450A1 (en) Engineered constructs with cassette arrangements for increased transcription of rna payloads
HK40049102A (en) Compositions and methods for treating duchenne muscular dystrophy

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40095055

Country of ref document: HK