JP2019524149A - 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 - Google Patents

一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Download PDF

Info

Publication number
JP2019524149A
JP2019524149A JP2019510339A JP2019510339A JP2019524149A JP 2019524149 A JP2019524149 A JP 2019524149A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019524149 A JP2019524149 A JP 2019524149A
Authority
JP
Japan
Prior art keywords
sequence
seq
snp
mutation
pam
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
JP2019510339A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019524149A5 (enExample
Inventor
タラ ムーア
タラ ムーア
アンドリュー ネスビット
アンドリュー ネスビット
デイビッド コートニー
デイビッド コートニー
ケイティー クリスティー
ケイティー クリスティー
ジーン リー
ジーン リー
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 JP2019524149A publication Critical patent/JP2019524149A/ja
Publication of JP2019524149A5 publication Critical patent/JP2019524149A5/ja
Priority to JP2021214009A priority Critical patent/JP2022046694A/ja
Priority to JP2024002942A priority patent/JP2024041905A/ja
Pending legal-status Critical Current

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4741—Keratin; Cytokeratin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/495—Transforming growth factor [TGF]
    • 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
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Environmental Sciences (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Analytical Chemistry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Reproductive Health (AREA)
JP2019510339A 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Pending JP2019524149A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021214009A JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662377586P 2016-08-20 2016-08-20
US62/377,586 2016-08-20
US201762462808P 2017-02-23 2017-02-23
US62/462,808 2017-02-23
US201762501750P 2017-05-05 2017-05-05
US62/501,750 2017-05-05
PCT/US2017/047861 WO2018039145A1 (en) 2016-08-20 2017-08-21 Single guide rna, crispr/cas9 systems, and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021214009A Division JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Publications (2)

Publication Number Publication Date
JP2019524149A true JP2019524149A (ja) 2019-09-05
JP2019524149A5 JP2019524149A5 (enExample) 2020-10-01

Family

ID=61246315

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019510339A Pending JP2019524149A (ja) 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2021214009A Pending JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2021214009A Pending JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Country Status (6)

Country Link
US (1) US20190185850A1 (enExample)
EP (1) EP3500677A4 (enExample)
JP (3) JP2019524149A (enExample)
KR (2) KR102594051B1 (enExample)
CN (1) CN109963945A (enExample)
WO (1) WO2018039145A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPWO2018225807A1 (ja) * 2017-06-07 2020-04-09 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
JP2022046694A (ja) * 2016-08-20 2022-03-23 アベリノ ラボ ユーエスエー インコーポレイテッド 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
US11987809B2 (en) 2015-11-13 2024-05-21 Avellino Lab Usa, Inc. Methods for the treatment of corneal dystrophies

Families Citing this family (53)

* 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
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
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
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
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US12214056B2 (en) 2016-07-19 2025-02-04 Duke University Therapeutic applications of CPF1-based genome editing
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2020225754A1 (en) * 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
WO2020046861A1 (en) * 2018-08-27 2020-03-05 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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
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
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
GB2575930A (en) 2017-03-23 2020-01-29 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
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)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019183150A1 (en) * 2018-03-19 2019-09-26 Casebia Therapeutics Limited Liability Partnership Novel rna-programmable endonuclease systems and uses thereof
WO2019215216A1 (en) * 2018-05-08 2019-11-14 Katholieke Universiteit Leuven Biosensor
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CA3121191A1 (en) 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110551763B (zh) * 2019-08-08 2023-03-10 复旦大学 CRISPR/SlutCas9基因编辑系统及其应用
CN110499335B (zh) * 2019-08-08 2023-03-28 复旦大学 CRISPR/SauriCas9基因编辑系统及其应用
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
CN115515968A (zh) * 2019-12-11 2022-12-23 阿维利诺美国实验室股份有限公司 使用短干扰RNA对具有R124H突变的转化生长因子β诱导基因的等位基因特异性沉默
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111926015B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
WO2022170059A1 (en) * 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2023034704A1 (en) * 2021-08-31 2023-03-09 The Regents Of The University Of California Products and methods for annotating gene function using locally haploid, human non-cancer cells
WO2023147428A2 (en) * 2022-01-26 2023-08-03 Orthobio Therapeutics, Inc. Gene editing to improve joint function
EP4463555A1 (en) * 2022-02-25 2024-11-20 Incisive Genetics, Inc. Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors
CN115948537B (zh) * 2022-12-19 2024-04-09 湖南家辉生物技术有限公司 一种基因chst3复合杂合突变的应用及检测试剂和应用
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
WO2025043140A1 (en) * 2023-08-23 2025-02-27 Emendobio Inc. Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele
CN119464347B (zh) * 2024-11-26 2025-10-03 扬州大学 基于CRISPR-Cas9系统的噬菌体基因组编辑载体及其编辑方法和应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
JP2016502840A (ja) * 2012-12-06 2016-02-01 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ Ｃｒｉｓｐｒに基づくゲノム修飾および制御

Family Cites Families (24)

* 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
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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
RS60838B1 (sr) 2012-08-29 2020-10-30 Sangamo Therapeutics Inc Postupci i kompozicije za tretman genskog stanja
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
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
DK2921557T3 (en) 2012-12-12 2016-11-07 Broad Inst Inc Design of systems, methods and optimized sequence manipulation guide compositions
IL307735A (en) 2012-12-12 2023-12-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
PL2931898T3 (pl) 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
EP2975117B1 (en) * 2013-03-11 2018-01-10 JCR Pharmaceuticals Co., Ltd. Method for producing human corneal epithelium sheet
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
AU2014281026B2 (en) * 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
IL303973A (en) * 2013-07-09 2023-08-01 Harvard College Multiplexed RNA-guided genome engineering
US20150044772A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
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
AU2016335572B2 (en) * 2015-10-09 2022-12-08 The Children's Hospital Of Philadelphia Compositions and methods for treating Huntington's disease and related disorders
WO2017083852A1 (en) * 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies
KR102594051B1 (ko) * 2016-08-20 2023-10-26 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
JP2016502840A (ja) * 2012-12-06 2016-02-01 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ Ｃｒｉｓｐｒに基づくゲノム修飾および制御

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GENE THERAPY, vol. 23, JPN6021029995, August 2015 (2015-08-01), pages 108 - 112, ISSN: 0004958737 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11987809B2 (en) 2015-11-13 2024-05-21 Avellino Lab Usa, Inc. Methods for the treatment of corneal dystrophies
JP2022046694A (ja) * 2016-08-20 2022-03-23 アベリノ ラボ ユーエスエー インコーポレイテッド 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JPWO2018225807A1 (ja) * 2017-06-07 2020-04-09 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
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

Also Published As

Publication number Publication date
CN109963945A (zh) 2019-07-02
JP2022046694A (ja) 2022-03-23
JP2024041905A (ja) 2024-03-27
WO2018039145A9 (en) 2018-05-03
WO2018039145A1 (en) 2018-03-01
US20190185850A1 (en) 2019-06-20
KR20190041499A (ko) 2019-04-22
EP3500677A1 (en) 2019-06-26
KR20230155013A (ko) 2023-11-09
KR102594051B1 (ko) 2023-10-26
EP3500677A4 (en) 2020-04-01

Similar Documents

Publication Publication Date Title
JP2024041905A (ja) 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP7239649B2 (ja) 角膜ジストロフィーの治療方法
Giannelli et al. Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9. PHP. B-based delivery
CN110300803B (zh) 提高细胞基因组中同源定向修复(hdr)效率的方法
JP2023054185A (ja) Ｄｎａが編集された真核細胞を製造する方法、および当該方法に用いられるキット
US20190330603A1 (en) Crispr-cas system, materials and methods
JPWO2019160077A1 (ja) 低抗原性細胞の製造方法
WO2020046861A1 (en) Crispr/cas9 systems, and methods of use thereof
US20210032612A1 (en) CRISPR/Cas9 Systems, and Methods of Use Thereof
WO2019017321A1 (ja) 遺伝子変異導入方法
US20220213488A1 (en) Correction of the two most prevalent ush2a mutations by genome editing
WO2020225754A1 (en) Crispr gene editing for autosomal dominant diseases
WO2022072458A1 (en) Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
US20210222171A1 (en) Crispr/cas9 systems, and methods of use thereof
US20220265863A1 (en) Compositions and methods for the treatment of dba using gata1 gene therapy
US20230407279A1 (en) Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
Neiteler Establishing a human cell-based model system for macular degeneration
HK1259885A1 (en) Methods for the treatment of corneal dystrophies
WO2023044510A2 (en) Crispr gene editing for diseases associated with a gene mutation or single-nucleotide polymorphism (snp)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20190423

RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20190423

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200820

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200820

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210803

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211102

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211228

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220426

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220720

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230110