JP6929865B2 - 角膜ジストロフィーの治療方法 - Google Patents

角膜ジストロフィーの治療方法 Download PDF

Info

Publication number
JP6929865B2
JP6929865B2 JP2018545108A JP2018545108A JP6929865B2 JP 6929865 B2 JP6929865 B2 JP 6929865B2 JP 2018545108 A JP2018545108 A JP 2018545108A JP 2018545108 A JP2018545108 A JP 2018545108A JP 6929865 B2 JP6929865 B2 JP 6929865B2
Authority
JP
Japan
Prior art keywords
nucleic acid
certain embodiments
corneal
stem cells
target nucleic
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.)
Expired - Fee Related
Application number
JP2018545108A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018534114A5 (cg-RX-API-DMAC7.html
JP2018534114A (ja
Inventor
アンドリュー ネスビット
アンドリュー ネスビット
ジーン リー
ジーン リー
スン−ヨン チョ
スン−ヨン チョ
ラリー デディオニシオ
ラリー デディオニシオ
タラ ムーア
タラ ムーア
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.)
Avellino Lab USA Inc
Original Assignee
Avellino Lab USA 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 Avellino Lab USA Inc filed Critical Avellino Lab USA Inc
Publication of JP2018534114A publication Critical patent/JP2018534114A/ja
Publication of JP2018534114A5 publication Critical patent/JP2018534114A5/ja
Priority to JP2021131116A priority Critical patent/JP7239649B2/ja
Application granted granted Critical
Publication of JP6929865B2 publication Critical patent/JP6929865B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0621Eye cells, e.g. cornea, iris pigmented cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/3604Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix characterised by the human or animal origin of the biological material, e.g. hair, fascia, fish scales, silk, shellac, pericardium, pleura, renal tissue, amniotic membrane, parenchymal tissue, fetal tissue, muscle tissue, fat tissue, enamel
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/3641Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix characterised by the site of application in the body
    • A61L27/3666Epithelial tissues other than skin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/3683Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix subjected to a specific treatment prior to implantation, e.g. decellularising, demineralising, grinding, cellular disruption/non-collagenous protein removal, anti-calcification, crosslinking, supercritical fluid extraction, enzyme treatment
    • A61L27/3695Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix subjected to a specific treatment prior to implantation, e.g. decellularising, demineralising, grinding, cellular disruption/non-collagenous protein removal, anti-calcification, crosslinking, supercritical fluid extraction, enzyme treatment characterised by the function or physical properties of the final product, where no specific conditions are defined to achieve this
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/50Materials characterised by their function or physical properties, e.g. injectable or lubricating compositions, shape-memory materials, surface modified materials
    • A61L27/54Biologically active materials, e.g. therapeutic substances
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • 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
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L2430/00Materials or treatment for tissue regeneration
    • A61L2430/16Materials or treatment for tissue regeneration for reconstruction of eye parts, e.g. intraocular lens, cornea
    • 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
    • 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
    • C12N2510/00Genetically modified 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
    • C12N2533/00Supports or coatings for cell culture, characterised by material
    • C12N2533/90Substrates of biological origin, e.g. extracellular matrix, decellularised tissue
    • C12N2533/92Amnion; Decellularised dermis or mucosa
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Oral & Maxillofacial Surgery (AREA)
  • Transplantation (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
JP2018545108A 2015-11-13 2016-11-14 角膜ジストロフィーの治療方法 Expired - Fee Related JP6929865B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021131116A JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255310P 2015-11-13 2015-11-13
US62/255,310 2015-11-13
PCT/US2016/061893 WO2017083852A1 (en) 2015-11-13 2016-11-14 Methods for the treatment of corneal dystrophies

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021131116A Division JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

Publications (3)

Publication Number Publication Date
JP2018534114A JP2018534114A (ja) 2018-11-22
JP2018534114A5 JP2018534114A5 (cg-RX-API-DMAC7.html) 2019-12-19
JP6929865B2 true JP6929865B2 (ja) 2021-09-01

Family

ID=58696178

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018545108A Expired - Fee Related JP6929865B2 (ja) 2015-11-13 2016-11-14 角膜ジストロフィーの治療方法
JP2021131116A Active JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2021131116A Active JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

Country Status (7)

Country Link
US (1) US11987809B2 (cg-RX-API-DMAC7.html)
EP (2) EP3374502B1 (cg-RX-API-DMAC7.html)
JP (2) JP6929865B2 (cg-RX-API-DMAC7.html)
KR (1) KR20180120670A (cg-RX-API-DMAC7.html)
CN (1) CN108699542A (cg-RX-API-DMAC7.html)
ES (1) ES2905558T3 (cg-RX-API-DMAC7.html)
WO (1) WO2017083852A1 (cg-RX-API-DMAC7.html)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
KR20230155013A (ko) * 2016-08-20 2023-11-09 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
AU2018280116B2 (en) * 2017-06-05 2025-03-27 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
JP7161730B2 (ja) * 2017-06-07 2022-10-27 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019060469A2 (en) * 2017-09-19 2019-03-28 Massachusetts Institute Of Technology CAS9 STREPTOCOCCUS CANIS AS A GENOMIC ENGINEERING PLATFORM WITH NEW PAM SPECIFICITY
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
EP3690047A4 (en) * 2017-09-28 2021-11-03 Toolgen Incorporated ARTIFICIAL GENOME MANIPULATION FOR GENE EXPRESSION REGULATION
EP3724332A1 (en) * 2017-12-14 2020-10-21 CRISPR Therapeutics AG Novel rna-programmable endonuclease systems and their use in genome editing and other applications
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
SG11202010260XA (en) 2018-04-25 2020-11-27 Childrens Medical Center Abcb5 ligands and substrates
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN113646434B (zh) * 2018-12-20 2023-05-30 北京大学 使用加标签的向导rna构建体进行高效基因筛选的组合物和方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001306T5 (de) * 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
AU2020248464A1 (en) * 2019-03-28 2021-10-14 Children's Medical Center Corporation Highly functional manufactured ABCB5+ mesenchymal stem cells
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4065702A4 (en) * 2019-11-27 2024-03-20 The General Hospital Corporation SYSTEM AND METHOD FOR ACTIVATING GENE EXPRESSION
RU2730975C1 (ru) * 2020-02-18 2020-08-26 Ирина Викторовна Булыгина Способ лечения эндотелиально-эпителиальной дистрофии роговицы
WO2023004189A1 (en) * 2021-07-23 2023-01-26 Avellino Lab Usa, Inc. Method for detecting alleles associated with keratoconus
CN116286823A (zh) * 2023-04-04 2023-06-23 尧唐(上海)生物科技有限公司 靶向TGFBI基因的sgRNA、载体及其应用

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
US5925517A (en) 1993-11-12 1999-07-20 The Public Health Research Institute Of The City Of New York, Inc. Detectably labeled dual conformation oligonucleotide probes, assays and kits
CA2591550C (en) 1996-06-04 2009-04-28 University Of Utah Research Foundation Monitoring hybridization during pcr
US6117635A (en) 1996-07-16 2000-09-12 Intergen Company Nucleic acid amplification oligonucleotides with molecular energy transfer labels and methods based thereon
US6018713A (en) 1997-04-09 2000-01-25 Coli; Robert D. Integrated system and method for ordering and cumulative results reporting of medical tests
JP3380744B2 (ja) 1998-05-19 2003-02-24 株式会社日立製作所 センサおよびこれを利用した測定装置
GB9812768D0 (en) 1998-06-13 1998-08-12 Zeneca Ltd Methods
US6171112B1 (en) 1998-09-18 2001-01-09 Wyngate, Inc. Methods and apparatus for authenticating informed consent
AU4135600A (en) 1999-03-29 2000-10-16 Genset Prostate cancer associated human fibronectin gene and biallelic markers
US6270970B1 (en) 1999-05-14 2001-08-07 Promega Corporation Mixed-bed solid phase and its use in the isolation of nucleic acids
ATE381626T1 (de) 2000-04-13 2008-01-15 Univ Georgetown Genetische diagnose zur feststellung von qt- verlängerungen als unerwünschte reaktion auf arzneimittel
US8438042B2 (en) 2002-04-25 2013-05-07 National Biomedical Research Foundation Instruments and methods for obtaining informed consent to genetic tests
US20030176650A1 (en) 2001-08-09 2003-09-18 Grosse William M. Nucleic acids, polypeptides, single nucleotide polymorphisms and methods of use thereof
WO2003046526A1 (de) 2001-11-28 2003-06-05 Graffinity Pharmaceuticals Ag Spr-sensorflächenträger
US6943417B2 (en) 2003-05-01 2005-09-13 Clemson University DNA-based memory device and method of reading and writing same
US20050019757A1 (en) 2003-06-12 2005-01-27 Stolarchuk Danylo J. Contaminant detection apparatus
WO2005015198A1 (ja) 2003-08-06 2005-02-17 Nippon Telegraph And Telephone Corporation 多孔質材料を用いた分子の検出方法ならびに該多孔質材料及び該多孔質材料の製造方法
CA2544129A1 (en) 2003-10-22 2005-05-06 The Regents Of The University Of California Methods for preparing and functionalizing nanoparticles
WO2005047534A2 (en) 2003-10-28 2005-05-26 Bayer Healthcare Ag Methods and compositions for the response prediction of malig-nant neoplasia to treatment
EP1541528A1 (en) 2003-12-08 2005-06-15 Institut Jozef Stefan Quasi-one-dimensional polymers based on the metal-chalcogen-halogen system
US20060057604A1 (en) 2004-03-15 2006-03-16 Thinkfar Nanotechnology Corporation Method for electrically detecting oligo-nucleotides with nano-particles
MXPA06013440A (es) 2004-05-19 2007-06-12 Vp Holding Llc Sensor optico con estructura estratificada de plasmon para deteccion mejorada de grupos quimicos por dispersion raman intensificada por superficie.
US7713849B2 (en) 2004-08-20 2010-05-11 Illuminex Corporation Metallic nanowire arrays and methods for making and using same
US7332329B2 (en) 2004-09-24 2008-02-19 Wisconsin Alumni Research Foundation Versatile substrate for SPR detection
WO2006096011A1 (en) 2005-03-08 2006-09-14 Medigenes Co., Ltd. PHARMACEUTICAL COMPOSITION FOR TREATING AVELLINO CORNEA DYSTROPHY COMPRISING AN ANTIBODY AGAINST TGF-β
JP2006250668A (ja) 2005-03-10 2006-09-21 Tatsuro Endo 非標識バイオチップ
EP1715326A1 (en) 2005-04-22 2006-10-25 Universität Heidelberg Sensor chip with connected non-metallic particles comprising a metallic coating
WO2007002567A2 (en) 2005-06-23 2007-01-04 Nanosphere, Inc. Selective isolation and concentration of nucleic acids from complex samples
KR20070076532A (ko) 2006-01-18 2007-07-24 메디제네스(주) 각막이영양증 진단용 dna 칩
CN101374850A (zh) 2006-01-18 2009-02-25 株式会社美迪基尼斯 用于角膜营养不良诊断的dna芯片
WO2007109702A2 (en) 2006-03-21 2007-09-27 Washington State University Research Foundation Genetic polymorphisms in the corticotropin-releasing hormone(crh) gene as markers for improving beef marbling score and/or subcutaneous fat depth
EP1932922A1 (de) 2006-12-13 2008-06-18 Desitin Arzneimittel GmbH Schnelltest zum Nachweis von DNA-Sequenzen
EP2115123A4 (en) 2007-01-16 2010-07-07 Applied Biosystems Llc SELECTIVE LYSE OF SPERM CELLS
US7898658B2 (en) 2007-01-23 2011-03-01 The Regents Of The University Of California Platform for chemical and biological sensing by surface-enhanced Raman spectroscopy
KR100891096B1 (ko) 2007-02-13 2009-03-31 삼성전자주식회사 올리고머 프로브 어레이 및 이의 제조 방법
JP5222599B2 (ja) 2007-07-20 2013-06-26 株式会社日立ハイテクノロジーズ 核酸分析デバイス及びそれを用いた核酸分析装置
CN101144812B (zh) 2007-10-17 2012-02-22 中国科学院光电技术研究所 一种局域表面等离子体生化传感器的制作方法
US20120231537A1 (en) 2008-04-30 2012-09-13 Gradalis, Inc. Highly Pure Plasmid DNA Preparations
KR101251538B1 (ko) 2009-04-17 2013-04-08 (주)아벨리노 아벨리노 각막이상증 진단용 프라이머
US8865402B2 (en) 2009-08-26 2014-10-21 Clemson University Research Foundation Nanostructured substrates for surface enhanced raman spectroscopy (SERS) and detection of biological and chemical analytes by electrical double layer (EDL) capacitance
KR101125212B1 (ko) 2010-10-01 2012-03-21 (주)아벨리노 아벨리노 각막이상증 진단용 시스템
KR20130027281A (ko) 2011-09-07 2013-03-15 (주)에스엘에스 원추각막 검사 방법 및 키트
US9895451B2 (en) 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
AU2013308770B2 (en) 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
EP2921557B1 (en) 2012-12-12 2016-07-13 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
ES2786193T3 (es) 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR102385934B1 (ko) * 2013-02-19 2022-04-13 칠드런'즈 메디컬 센터 코포레이션 안구 질환을 치료하기 위한 abcb5(+) 줄기 세포
EP2975117B1 (en) 2013-03-11 2018-01-10 JCR Pharmaceuticals Co., Ltd. Method for producing human corneal epithelium sheet
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
KR20160029112A (ko) 2013-07-09 2016-03-14 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 멀티플렉스 rna-가이드된 게놈 조작
WO2015021426A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
EP3872192A1 (en) * 2013-11-15 2021-09-01 Avellino Lab USA, Inc. Methods for multiplex detection of alleles associated with ophthalmic conditions
AU2014361781B2 (en) * 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
US20150225801A1 (en) * 2014-02-11 2015-08-13 California Institute Of Technology Recording and mapping lineage information and molecular events in individual cells
SG10201806498RA (en) * 2014-06-27 2018-08-30 Univ California Cultured mammalian limbal stem cells, methods for generating the same, and uses thereof
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
GB2531454A (en) 2016-01-10 2016-04-20 Snipr Technologies Ltd Recombinogenic nucleic acid strands in situ
MX2017016921A (es) * 2015-07-02 2018-04-10 Univ Johns Hopkins Tratamientos basados en crispr / cas9.
WO2017062983A1 (en) 2015-10-09 2017-04-13 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2020046861A1 (en) 2018-08-27 2020-03-05 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
US20210222171A1 (en) 2016-08-20 2021-07-22 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
WO2019165322A1 (en) 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
KR20230155013A (ko) 2016-08-20 2023-11-09 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법
US20220056440A1 (en) 2016-08-20 2022-02-24 Tara Moore Crispr gene editing for autosomal dominant diseases
CN106282383B (zh) 2016-10-08 2019-05-31 山东省眼科研究所 Krt12基因在检测圆锥角膜中的应用
MX2019006157A (es) 2016-11-28 2019-11-21 Univ Texas Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.
WO2018200980A1 (en) 2017-04-28 2018-11-01 Avellino Lab Usa, Inc. Methods for detecting alleles associated with keratoconus
EP3676378A4 (en) 2017-08-31 2021-06-09 The New York Genome Center PROCEDURES AND COMPOSITIONS USING CRIMP-CPF1 AND PAIRED GUIDE-CRISPR-RNA FOR PROGRAMMABLE GENOMIC DELETIONS

Also Published As

Publication number Publication date
WO2017083852A1 (en) 2017-05-18
EP3374502B1 (en) 2021-10-27
US11987809B2 (en) 2024-05-21
EP3374502A4 (en) 2019-07-10
ES2905558T3 (es) 2022-04-11
EP3374502A1 (en) 2018-09-19
JP2018534114A (ja) 2018-11-22
WO2017083852A8 (en) 2018-05-31
US20190085288A1 (en) 2019-03-21
CN108699542A (zh) 2018-10-23
JP2021183626A (ja) 2021-12-02
JP7239649B2 (ja) 2023-03-14
EP4036228A1 (en) 2022-08-03
KR20180120670A (ko) 2018-11-06

Similar Documents

Publication Publication Date Title
JP7239649B2 (ja) 角膜ジストロフィーの治療方法
JP2022046694A (ja) 一本鎖ガイドRNA、CRISPR/Cas9システム、及びそれらの使用方法
Giannelli et al. Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9. PHP. B-based delivery
CN110248957B (zh) 经人工操纵的sc功能控制系统
Kim et al. Generation of TGFBI knockout ABCG2+/ABCB5+ double-positive limbal epithelial stem cells by CRISPR/Cas9-mediated genome editing
EP3636754A1 (en) Gene therapy for granular corneal dystrophy
JP7393770B2 (ja) ゲノム編集による2つの最も高頻度に見られるush2a突然変異の修正
WO2022072458A1 (en) Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
KR101658135B1 (ko) 혈액 응고인자 ⅷ 유전자를 타겟으로 하는 엔도뉴클레아제 및 이의 용도
US20230407279A1 (en) Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
Shen et al. Association of a chromosomal rearrangement event with mouse posterior polymorphous corneal dystrophy and alterations in Csrp2bp, Dzank1, and Ovol2 gene expression
CN115427573A (zh) 用于治疗常染色体显性best1相关视网膜病的组合物
WO2021171688A1 (ja) 遺伝子ノックイン方法、遺伝子ノックイン細胞作製方法、遺伝子ノックイン細胞、がん化リスク評価方法、がん細胞製造方法、及びこれらに用いるためのキット
HK1259885A1 (en) Methods for the treatment of corneal dystrophies
JP2018042503A (ja) 標的遺伝子の標識方法、ベクターシステム、細胞、及び遺伝子活性維持標的遺伝子編集キット
Ivanenko et al. Genome Editing in Therapy of Genodermatoses
Kowalczyk et al. Genetics and Gene Therapy of the TGFBI Corneal Dystrophies
Kayser Characterization of zebrafish lines with mutations in the nephronophthisis-associated genes nphp1, nphp2, nphp4 and nphp8
Giacalone Treating retinal disease
Lemanski et al. Cellular, molecular, and developmental studies on heart development in normal and cardiac mutant axolotls, Ambystoma mexicanum
Bonafont Aragó et al. Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing
Messineo Development of a gene targeting strategy (Recombinase-Mediated CAssette Exchange) to generate cellular models of MYH9-related disease

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191108

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20191108

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20201104

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210203

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210309

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210609

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20210713

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20210811

R150 Certificate of patent or registration of utility model

Ref document number: 6929865

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees