SG10201710487VA - Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components - Google Patents

Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components

Info

Publication number
SG10201710487VA
SG10201710487VA SG10201710487VA SG10201710487VA SG10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA
Authority
SG
Singapore
Prior art keywords
crispr
diseases
delivery
compositions
therapeutic applications
Prior art date
Application number
SG10201710487VA
Other languages
English (en)
Inventor
Zhang Feng
Cong Le
Ran Fei
HEIDENREICH Matthias
Swiech Lukasz
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Harvard College
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52105124&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=SG10201710487V(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/US2013/074667 external-priority patent/WO2014093622A2/en
Application filed by Broad Inst Inc, Massachusetts Inst Technology, Harvard College filed Critical Broad Inst Inc
Publication of SG10201710487VA publication Critical patent/SG10201710487VA/en

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Environmental Sciences (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Mycology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG10201710487VA 2013-06-17 2014-06-11 Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components SG10201710487VA (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US201361862355P 2013-08-05 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US201361915225P 2013-12-12 2013-12-12
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US201461979879P 2014-04-15 2014-04-15

Publications (1)

Publication Number Publication Date
SG10201710487VA true SG10201710487VA (en) 2018-01-30

Family

ID=52105124

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201510286QA SG11201510286QA (en) 2013-06-17 2014-06-11 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
SG10201710487VA SG10201710487VA (en) 2013-06-17 2014-06-11 Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG11201510286QA SG11201510286QA (en) 2013-06-17 2014-06-11 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components

Country Status (14)

Country Link
US (2) US10946108B2 (enExample)
EP (2) EP3597755A1 (enExample)
JP (1) JP6702858B2 (enExample)
KR (2) KR20160056869A (enExample)
CN (2) CN105793425B (enExample)
AU (1) AU2014281031B2 (enExample)
BR (1) BR122021009076B1 (enExample)
CA (1) CA2915795C (enExample)
IL (1) IL243195B (enExample)
MX (1) MX374532B (enExample)
RU (1) RU2716421C2 (enExample)
SG (2) SG11201510286QA (enExample)
WO (1) WO2014204729A1 (enExample)
ZA (1) ZA201508961B (enExample)

Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11643652B2 (en) 2019-03-19 2023-05-09 The Broad Institute, Inc. Methods and compositions for prime editing nucleotide sequences
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
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)
US11932884B2 (en) 2017-08-30 2024-03-19 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US12473573B2 (en) 2013-09-06 2025-11-18 President And Fellows Of Harvard College Switchable Cas9 nucleases and uses thereof
US12509680B2 (en) 2023-05-31 2025-12-30 The Broad Institute, Inc. Methods and compositions for prime editing nucleotide sequences

Families Citing this family (315)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011140441A2 (en) 2010-05-06 2011-11-10 Children's Hospital Medical Center Methods and systems for converting precursor cells into intestinal tissues through directed differentiation
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
WO2013139861A1 (en) 2012-03-20 2013-09-26 Luc Montagnier Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014065596A1 (en) 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
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
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CA2894668A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
RS62263B1 (sr) 2013-04-16 2021-09-30 Regeneron Pharma Ciljana modifikacija genoma pacova
KR20160056869A (ko) 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
DK3011029T3 (da) 2013-06-17 2020-03-16 Broad Inst Inc Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015040402A1 (en) 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
RU2725520C2 (ru) 2013-12-11 2020-07-02 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
KR20160097327A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 ｃａｓ 효소의 발현의 변경을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 방법
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
KR20250068794A (ko) 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
CA2932479A1 (en) 2013-12-12 2015-06-18 The Rockefeller University Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CN106103705A (zh) 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
EP3514246B1 (en) 2014-02-27 2021-11-17 The Broad Institute, Inc. T cell balance gene expression and methods of use thereof
EP3149156B1 (en) 2014-05-28 2021-02-17 Children's Hospital Medical Center Methods and systems for converting precursor cells into gastric tissues through directed differentiation
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
WO2016049251A1 (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 mutations in leukocytes
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
KR20170070136A (ko) 2014-10-15 2017-06-21 리제너론 파마슈티칼스 인코포레이티드 만능성 세포를 생성하거나 유지하는 방법 및 조성물
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
IL301900A (en) 2014-11-21 2023-06-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
EP3626832B1 (en) 2014-11-25 2024-06-12 The Brigham and Women's Hospital, Inc. Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease
AU2015360502A1 (en) 2014-12-10 2017-06-29 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2016100974A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
CN104611368B (zh) * 2015-01-15 2018-05-25 中国科学院广州生物医药与健康研究院 重组后不产生移码突变的载体、在爪蛙基因组中进行基因定点敲入的方法及应用
EP3262193A2 (en) 2015-02-26 2018-01-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
EP3271460A4 (en) * 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1)
GB2536650A (en) 2015-03-24 2016-09-28 Augmedics Ltd Method and system for combining video-based and optic-based augmented reality in a near eye display
JP6851319B2 (ja) * 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
CN107849547B (zh) * 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
WO2016196539A2 (en) 2015-06-01 2016-12-08 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
FI3430134T3 (fi) 2015-06-18 2023-01-13 Uusia CRISPR-entsyymejä ja järjestelmiä
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
EP4545544A3 (en) 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017023803A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Modified cells and methods of therapy
WO2017024319A1 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Tunable endogenous protein degradation
EP3334484B1 (en) 2015-08-12 2024-12-25 The General Hospital Corporation Compositions that promote hypoxia or the hypoxia response for the treatment and prevention of mitochondrial dysfunction
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2017048466A1 (en) * 2015-09-15 2017-03-23 The Regents Of The University Of California Compositions and methods for delivering biotherapeutics
WO2017053879A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
KR20250021623A (ko) 2015-10-22 2025-02-13 더 브로드 인스티튜트, 인코퍼레이티드 타입 vi-b crispr 효소 및 시스템
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
US11092607B2 (en) 2015-10-28 2021-08-17 The Board Institute, Inc. Multiplex analysis of single cell constituents
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
AU2016355178B9 (en) 2015-11-19 2019-05-30 Massachusetts Institute Of Technology Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity
CN109072195A (zh) 2015-12-30 2018-12-21 诺华股份有限公司 具有增强功效的免疫效应细胞疗法
CN109312335B (zh) 2016-01-11 2023-10-24 斯坦福大学托管董事会 嵌合蛋白和调节基因表达的方法
CN108463229B (zh) 2016-01-11 2023-10-17 斯坦福大学托管董事会 嵌合蛋白和免疫治疗方法
SG11201806134SA (en) 2016-01-27 2018-08-30 Oncorus Inc Oncolytic viral vectors and uses thereof
JP7542298B2 (ja) * 2016-02-12 2024-08-30 コンビジーン エービー ベクター
EP3420102B1 (en) 2016-02-22 2024-04-03 Massachusetts Institute of Technology Methods for identifying and modulating immune phenotypes
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
JP7324583B2 (ja) 2016-05-20 2023-08-10 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法
WO2017219027A1 (en) 2016-06-17 2017-12-21 The Broad Institute Inc. Type vi crispr orthologs and systems
EP4282478A3 (en) 2016-06-21 2024-03-06 The Curators of the University of Missouri Modified dystrophin proteins
EP3481431A4 (en) * 2016-07-05 2020-01-01 The Johns Hopkins University CRISPR / CAS9 COMPOSITIONS AND METHODS FOR TREATING CANCER
MA45670A (fr) 2016-07-13 2019-05-22 Vertex Pharma Procédés, compositions et kits pour augmenter l'efficacité d'édition du génome
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CN110312799A (zh) * 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
EP4485466A3 (en) * 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US11630103B2 (en) 2016-08-17 2023-04-18 The Broad Institute, Inc. Product and methods useful for modulating and evaluating immune responses
AU2017313912B2 (en) * 2016-08-19 2024-01-04 Whitehead Institute For Biomedical Research Methods of editing DNA methylation
JP7050215B2 (ja) * 2016-08-19 2022-04-08 ツールゲン インコーポレイテッド 人工的に操作された血管新生調節系
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
WO2018053053A1 (en) 2016-09-13 2018-03-22 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
KR102546194B1 (ko) 2016-11-04 2023-06-21 칠드런즈 호스피탈 메디칼 센터 간 유사 장기 조성물 및 이를 제조 및 사용하는 방법
WO2018089386A1 (en) 2016-11-11 2018-05-17 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action
US12404514B2 (en) 2016-12-09 2025-09-02 The Broad Institute, Inc. CRISPR-systems for modifying a trait of interest in a plant
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018129203A2 (en) * 2017-01-06 2018-07-12 The Regents Of The University Of California Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
JP7215716B2 (ja) * 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
WO2018140644A1 (en) * 2017-01-25 2018-08-02 Musc Foundation For Research Development Modified t cells and uses thereof
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
CN110612113B (zh) * 2017-02-07 2024-03-26 加利福尼亚大学董事会 单倍体机能不全的基因疗法
JP7227912B2 (ja) 2017-02-08 2023-02-24 ダナ－ファーバー キャンサー インスティテュート，インコーポレイテッド キメラ抗原受容体の調節
CN110662838B (zh) * 2017-02-22 2024-05-28 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
US11932856B2 (en) * 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3595694A4 (en) * 2017-03-14 2021-06-09 The Regents of The University of California Engineering crispr cas9 immune stealth
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系
JP2020513815A (ja) * 2017-03-15 2020-05-21 ザ・ブロード・インスティテュート・インコーポレイテッド クラスター化短鎖反復回文配列エフェクター系に基づくウイルス検出用診断法
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
EP3600428A4 (en) * 2017-03-23 2020-08-26 Dnarx SYSTEMS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS IN VIVO
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
EP3606518A4 (en) 2017-04-01 2021-04-07 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTION AND MODULATION OF IMMUNOTHERAPY RESISTANCE GENE SIGNATURE IN CANCER
EP3610266A4 (en) 2017-04-12 2021-04-21 Massachusetts Eye and Ear Infirmary TUMOR SIGNATURE FOR METASTASIS, COMPOSITIONS OF MATERIAL AND THEIR METHODS OF USE
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
WO2018191673A1 (en) 2017-04-14 2018-10-18 Children's Hospital Medical Center Multi donor stem cell compositions and methods of making same
WO2018195019A1 (en) 2017-04-18 2018-10-25 The Broad Institute Inc. Compositions for detecting secretion and methods of use
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
WO2018201086A1 (en) 2017-04-28 2018-11-01 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
US12226479B2 (en) 2017-05-11 2025-02-18 The General Hospital Corporation Methods and compositions of use of CD8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
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
WO2018213278A1 (en) * 2017-05-15 2018-11-22 The Trustees Of Columbia University In The City Of New York Reprogramming metabolism by inhibiting vhl for treatment of neurodegeneration
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US11866697B2 (en) 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3406139A1 (en) 2017-05-26 2018-11-28 Medizinische Hochschule Hannover Method for genetically modifying a vascularised tissue
CA3102054A1 (en) 2017-06-05 2018-12-13 Fred Hutchinson Cancer Research Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
WO2018232195A1 (en) 2017-06-14 2018-12-20 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
WO2018232382A1 (en) * 2017-06-16 2018-12-20 Applied Stemcell, Inc. Gene editing methods with increased knock-in efficiency
EP3645054A4 (en) 2017-06-26 2021-03-31 The Broad Institute, Inc. CRISPR / CAS-ADENINE DESAMINASE COMPOSITIONS, TARGETED NUCLEIC ACID EDITING SYSTEMS AND METHODS
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
KR102007457B1 (ko) * 2017-06-30 2019-08-05 동국대학교 산학협력단 Cas9 단백질, 가이드 RNA 및 양친매성 펩타이드를 포함하는 나노복합체 및 이의 용도
WO2019002633A1 (en) 2017-06-30 2019-01-03 Cellectis CELLULAR IMMUNOTHERAPY FOR REPETITIVE ADMINISTRATION
WO2019003193A1 (en) 2017-06-30 2019-01-03 Novartis Ag METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS
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
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
WO2019018694A1 (en) 2017-07-19 2019-01-24 Dana-Farber Cancer Institute, Inc. DIAGNOSIS AND TREATMENT OF CANCER
CN109295054B (zh) * 2017-07-25 2024-02-06 广州普世利华科技有限公司 用于靶向病原体基因RNA的gRNA及基于C2c2的病原体基因的检测方法及试剂盒
US11612625B2 (en) 2017-07-26 2023-03-28 Oncorus, Inc. Oncolytic viral vectors and uses thereof
CA3073848A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
BR112020003609A2 (pt) 2017-09-29 2020-09-01 Regeneron Pharmaceuticals, Inc. sistema e método para formar uma emulsão
WO2019070755A1 (en) 2017-10-02 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
EP3694603A4 (en) 2017-10-10 2021-07-14 Children's Hospital Medical Center ESOPHAGUS TISSUE AND / OR ORGANOID COMPOSITIONS AND METHOD FOR MANUFACTURING THEREOF
CN109646678A (zh) * 2017-10-12 2019-04-19 中国科学院上海生命科学研究院 Sun2蛋白、其制药用途及药物
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
IL274082B1 (en) 2017-10-20 2025-09-01 Hutchinson Fred Cancer Res Systems and methods for producing genetically engineered B cells for expression of selected antibodies
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
CN109536494A (zh) * 2017-10-31 2019-03-29 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
MA50877A (fr) 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
US12458411B2 (en) 2017-12-07 2025-11-04 Augmedics Ltd. Spinous process clamp
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3727394A4 (en) 2017-12-21 2021-09-08 Children's Hospital Medical Center DIGITALIZED HUMAN ORGANOIDS AND METHOD OF USING THEREOF
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
KR20200110687A (ko) 2018-01-17 2020-09-24 버텍스 파마슈티칼스 인코포레이티드 유전체 편집 효율을 증가시키기 위한 퀴녹살리논 화합물, 조성물, 방법 및 키트
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
ES2980444T3 (es) 2018-01-17 2024-10-01 Vertex Pharma Inhibidores de ADN-PK
US11173216B2 (en) * 2018-01-18 2021-11-16 Wisconsin Alumni Research Foundation Gene editing-based method of attenuating the beta-amyloid pathway
BR112020018658A2 (pt) 2018-03-15 2020-12-29 KSQ Therapeutics, Inc. Composições de regulação gênica e métodos para imu-noterapia aprimorada
WO2019204585A1 (en) 2018-04-19 2019-10-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded dna
JP2021523696A (ja) * 2018-04-23 2021-09-09 デューク ユニバーシティ Ｄｎａメチル化の標的化された編集によるｓｎｃａ発現の下方調節
SI3560330T1 (sl) 2018-04-24 2022-08-31 KWS SAAT SE & Co. KGaA Rastline z izboljšano prebavljivostjo in markerskimi haplotipi
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
US12281301B2 (en) 2018-04-27 2025-04-22 The Broad Institute, Inc. Sequencing-based proteomics
EP3787543A4 (en) 2018-05-02 2022-01-19 Augmedics Ltd. REGISTRATION OF A FIDUCIAL MARKER FOR AN AUGMENTED REALITY SYSTEM
US20210386829A1 (en) 2018-05-04 2021-12-16 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US20210371932A1 (en) 2018-06-01 2021-12-02 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
US20210269866A1 (en) 2018-06-26 2021-09-02 The Broad Institute, Inc. Crispr effector system based amplification methods, systems, and diagnostics
CA3101066A1 (en) 2018-06-26 2020-01-02 The Broad Institute, Inc. Crispr/cas and transposase based amplification compositions, systems and methods
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
US20210214699A1 (en) * 2018-07-05 2021-07-15 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
JP7506657B2 (ja) 2018-07-26 2024-06-26 チルドレンズ ホスピタル メディカル センター 肝胆膵組織およびその作製方法
AU2019314433A1 (en) 2018-07-31 2021-03-25 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
WO2020033601A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
WO2020041387A1 (en) 2018-08-20 2020-02-27 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
EP3847650A1 (en) 2018-09-06 2021-07-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
US12378572B2 (en) 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
US12428622B2 (en) 2018-09-12 2025-09-30 Children's Hospital Medical Center Organoid compositions for the production of hematopoietic stem cells and derivatives thereof
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
KR20210070305A (ko) 2018-09-18 2021-06-14 브이엔브이 뉴코 인크. Arc 기반 캡시드 및 이의 용도
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020077236A1 (en) 2018-10-12 2020-04-16 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
CN111788302A (zh) * 2018-11-21 2020-10-16 南京北恒生物科技有限公司 修饰的t细胞及其用途
US11766296B2 (en) 2018-11-26 2023-09-26 Augmedics Ltd. Tracking system for image-guided surgery
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
JP2022514955A (ja) * 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
CA3133642A1 (en) * 2019-03-22 2020-10-01 Mayo Foundation For Medical Education And Research Materials and methods for treating polycystic kidney disease
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114096684B (zh) 2019-05-13 2024-11-19 科沃施种子欧洲股份两合公司 玉米的耐旱性
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
CA3140093A1 (en) * 2019-05-21 2020-11-26 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems
EP3972631B1 (en) * 2019-05-21 2024-07-10 Institut National de la Santé et de la Recherche Médicale (INSERM) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
EP3973059A4 (en) * 2019-05-22 2023-10-25 The University of North Carolina at Chapel Hill UBE3A GENES AND EXPRESSION CASSETTES AND THEIR USE
US20220218844A1 (en) * 2019-05-23 2022-07-14 Spacecraft Seven, Llc Gene therapy vectors for infantile malignant osteopetrosis
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
JP7610533B2 (ja) 2019-05-31 2025-01-08 チルドレンズ ホスピタル メディカル センター 造血幹細胞の生成および拡大方法
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CN113874510A (zh) 2019-06-04 2021-12-31 瑞泽恩制药公司 包括具有β滑移突变的人源化TTR基因座的非人动物和使用方法
WO2020264385A1 (en) * 2019-06-28 2020-12-30 Arizona Board Of Regents On Behalf Of The University Of Arizona Self-contained responsive biological systems and methods
CN114729383A (zh) 2019-07-02 2022-07-08 弗莱德哈钦森癌症研究中心 重组ad35载体及相关基因疗法改进
CN112168808B (zh) * 2019-07-02 2023-01-24 深圳市第二人民医院 一种基于crispr的细胞核靶向药物递送系统
CN114402071A (zh) * 2019-07-15 2022-04-26 新加坡国立大学 用于基因特异性脱甲基和激活的方法和组合物
US12178666B2 (en) 2019-07-29 2024-12-31 Augmedics Ltd. Fiducial marker
US11980506B2 (en) 2019-07-29 2024-05-14 Augmedics Ltd. Fiducial marker
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021041922A1 (en) 2019-08-30 2021-03-04 The Broad Institute, Inc. Crispr-associated mu transposase systems
WO2021044377A1 (en) 2019-09-05 2021-03-11 Crispr Therapeutics Ag Universal donor cells
US11118196B2 (en) * 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
EP4031660A1 (en) 2019-09-20 2022-07-27 The Broad Institute, Inc. Novel type vi crispr enzymes and systems
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4045522A1 (en) 2019-10-17 2022-08-24 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
CN112690409A (zh) * 2019-10-23 2021-04-23 冯生财 一种香辣肉条、肉片的配方及其制作方法
AU2020388387A1 (en) 2019-11-22 2022-06-02 President And Fellows Of Harvard College Ionic liquids for drug delivery
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US11891616B2 (en) 2019-12-05 2024-02-06 Board Of Regents Of The University Of Texas System Transgene cassettes designed to express a human MECP2 gene
US11382712B2 (en) 2019-12-22 2022-07-12 Augmedics Ltd. Mirroring in image guided surgery
AU2021207685A1 (en) * 2020-01-17 2022-08-25 Jumpcode Genomics, Inc. Methods of sample normalization
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
BR112022023443A2 (pt) 2020-05-29 2022-12-20 Kws Saat Se & Co Kgaa Indução de haploides em plantas
US11389252B2 (en) 2020-06-15 2022-07-19 Augmedics Ltd. Rotating marker for image guided surgery
US12239385B2 (en) 2020-09-09 2025-03-04 Augmedics Ltd. Universal tool adapter
KR102424351B1 (ko) * 2020-09-21 2022-08-05 한국과학기술원 파브알부민 유전자 발현 억제용 CRISPR/Cas9 시스템 및 이의 용도
CN114250228A (zh) * 2020-09-22 2022-03-29 广州瑞风生物科技有限公司 靶向CTGF基因的gRNA及其应用
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
WO2022144856A1 (en) 2020-12-31 2022-07-07 Crispr Therapeutics Ag Universal donor cells
CN113355360A (zh) * 2021-05-28 2021-09-07 上海碧博生物医药科技有限公司 一种gs基因敲除cho-k1细胞株的构建方法及悬浮细胞单克隆化
US11896445B2 (en) 2021-07-07 2024-02-13 Augmedics Ltd. Iliac pin and adapter
WO2023288086A2 (en) * 2021-07-16 2023-01-19 President And Fellows Of Harvard College Enhancers driving expression in motor neurons
US12150821B2 (en) 2021-07-29 2024-11-26 Augmedics Ltd. Rotating marker and adapter for image-guided surgery
CN118368974A (zh) 2021-07-30 2024-07-19 科沃施种子欧洲股份两合公司 具有提高的消化率和标记单倍型的植物
EP4387552A4 (en) 2021-08-18 2025-04-30 Augmedics Ltd. AUGMENTED REALITY SURGICAL SYSTEM USING DEPTH SENSING
WO2023059846A1 (en) 2021-10-08 2023-04-13 President And Fellows Of Harvard College Ionic liquids for drug delivery
CA3237300A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
US20240408236A1 (en) 2021-11-26 2024-12-12 Epigenic Therapeutics, Inc. Method of modulating pcsk9 and uses thereof
US11845079B2 (en) 2021-12-08 2023-12-19 International Business Machines Corporation Integrated silicon platform for electronic biosensors
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
CA3245130A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS RELATED TO ANGIOPOIETIN-TYPE PROTEIN 3 (ANGPTL3)
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023203521A1 (en) 2022-04-21 2023-10-26 Augmedics Ltd. Systems and methods for medical image visualization
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4577660A1 (en) 2022-08-26 2025-07-02 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
IL319523A (en) 2022-09-13 2025-05-01 Augmedics Ltd Augmented reality glasses for image-guided medical intervention
TW202432584A (zh) 2022-11-15 2024-08-16 美商嘉來克生命科學有限責任公司 抗papp-a抗體及其使用方法
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024193704A1 (en) * 2023-03-22 2024-09-26 Huidagene Therapeutics Co., Ltd. Guide nucleic acids targeting dmd and uses thereof
WO2024206911A2 (en) 2023-03-30 2024-10-03 Children's Hospital Medical Center Clinical-grade organoids
US20240352485A1 (en) 2023-04-19 2024-10-24 Flagship Pioneering Innovations Vii, Llc Compositions and methods for the production of libraries
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238977A2 (en) 2023-05-18 2024-11-21 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2024263961A2 (en) 2023-06-23 2024-12-26 Children's Hospital Medical Center Methods of matrix-free suspension culture
WO2025006419A1 (en) 2023-06-26 2025-01-02 Flagship Pioneering Innovations Vii, Llc Engineered plasmodia and related methods
CN116790603B (zh) * 2023-08-18 2023-10-31 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025072803A1 (en) 2023-09-29 2025-04-03 Children's Hospital Medical Center Ntrk2 signaling-mediated alveolar capillary injury and repair
WO2025212920A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Multi-zonal liver organoids
WO2025217202A1 (en) 2024-04-08 2025-10-16 Children's Hospital Medical Center Bile duct organoid
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

Family Cites Families (236)

* 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
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
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
EP0264166B1 (en) 1986-04-09 1996-08-21 Genzyme Corporation Transgenic animals secreting desired proteins into milk
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
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
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
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
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
JPH10510433A (ja) 1995-06-06 1998-10-13 アイシス・ファーマシューティカルス・インコーポレーテッド 高いキラル純度のホスホロチオエート結合を有するオリゴヌクレオチド
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
EP1025217B1 (en) 1997-10-24 2006-10-04 Invitrogen Corporation Recombinational cloning using nucleic acids having recombination sites
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7868149B2 (en) 1999-07-20 2011-01-11 Monsanto Technology Llc Plant genome sequence and uses thereof
US6479808B1 (en) 1999-07-07 2002-11-12 The United States Of America As Represented By The Administrator Of The National Aeronautics And Space Administration Method and systems for collecting data from multiple fields of view
US6603061B1 (en) 1999-07-29 2003-08-05 Monsanto Company Agrobacterium-mediated plant transformation method
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP3454818B1 (ja) 2001-03-16 2003-10-06 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
WO2002080851A2 (en) 2001-04-05 2002-10-17 The Johns Hopkins University Chimeric vaccines
EP1412371B1 (en) 2001-07-12 2016-02-24 University of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
JP2005509409A (ja) 2001-08-08 2005-04-14 ジェンザイム・コーポレーション 糖尿病および他の血糖疾患の治療方法
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
WO2003045304A2 (en) 2001-09-26 2003-06-05 Mayo Foundation For Medical Education And Research Mutable vaccines
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
US20090100536A1 (en) 2001-12-04 2009-04-16 Monsanto Company Transgenic plants with enhanced agronomic traits
JP2005512598A (ja) 2001-12-21 2005-05-12 オックスフォード バイオメディカ （ユーケー） リミテッド Ｅｉａｖなどのレンチウイルス発現ベクターを使用するトランスジェニック生物の作製方法
US7206639B2 (en) 2002-03-15 2007-04-17 Sarcos Investments Lc Cochlear drug delivery system and method
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
EP1532178A4 (en) 2002-06-11 2006-10-25 Scripps Research Inst Artificial transcription factors
ATE485031T1 (de) 2002-06-28 2010-11-15 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL BASED RNA INTERFERENCE AND ASSOCIATED METHODS AND COMPOSITIONS
AU2003290937A1 (en) * 2002-11-15 2004-06-15 Trustees Of Boston University Cis/trans riboregulators
US20060178297A1 (en) 2003-01-28 2006-08-10 Troy Carol M Systems and methods for silencing expression of a gene in a cell and uses thereof
US7601492B2 (en) 2003-07-03 2009-10-13 The Regents Of The University Of California Genome mapping of functional DNA elements and cellular proteins
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
AU2004263865B2 (en) 2003-08-08 2007-05-17 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
US8673634B2 (en) 2003-11-13 2014-03-18 Massachusetts Eye & Ear Infirmary Method for the treatment of hearing loss
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
WO2005072793A1 (en) 2004-01-29 2005-08-11 The Charles Stark Draper Laboratory, Inc. Implantable drug delivery apparatus
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
FR2872170B1 (fr) * 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
CN101277971A (zh) 2004-07-16 2008-10-01 美国政府健康及人类服务部 含cmv/r-核酸构建体的抗aids疫苗
US7715845B2 (en) * 2004-10-14 2010-05-11 Qualcomm Incorporated Tone hopping methods and apparatus
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
AU2006239169A1 (en) 2005-04-28 2006-11-02 Benitec, Limited. Multiple-RNAi expression cassettes for simultaneous delivery of RNAi agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
EP3284833B1 (en) * 2005-08-26 2021-12-01 DuPont Nutrition Biosciences ApS Use of crispr associated genes (cas)
WO2007030743A2 (en) 2005-09-08 2007-03-15 Massachusetts Eye & Ear Infirmary Cochlear implants containing biological cells and uses thereof
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
ES2377660T3 (es) 2005-10-28 2012-03-29 Mitsubishi Tanabe Pharma Corporation Nuevo péptido que penetra en las células
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
US9677123B2 (en) 2006-03-15 2017-06-13 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
WO2007134161A2 (en) 2006-05-11 2007-11-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the pcsk9 gene
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
AU2007320880A1 (en) 2006-11-14 2008-05-22 Cellectis Meganuclease variants cleaving a DNA target sequence from the HPRT gene and uses thereof
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
NZ579002A (en) 2007-03-02 2012-03-30 Danisco Cultures with improved phage resistance
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
WO2009019528A1 (en) 2007-08-03 2009-02-12 Cellectis Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof
ES2868136T3 (es) 2007-08-14 2021-10-21 Hutchinson Fred Cancer Res Disposición de matriz de agujas y método para administrar agentes terapéuticos
WO2009088786A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
EP2732819B1 (en) 2008-02-07 2019-10-16 Massachusetts Eye & Ear Infirmary Compounds that enhance Atoh-1 expression
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
ES2638448T3 (es) 2008-04-15 2017-10-20 Protiva Biotherapeutics Inc. Novedosas formulaciones de lípidos para la administración de ácidos nucleicos
JP2011523557A (ja) 2008-06-04 2011-08-18 メディカル リサーチ カウンシル ペプチド
EP2293800B1 (en) 2008-06-06 2016-10-05 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
GB0810912D0 (en) * 2008-06-13 2008-07-23 Inst Animal Health Ltd Vector
BRPI0910198A2 (pt) 2008-06-30 2016-01-12 Silenseed Ltd composição para administração a partir de um sítio local de um agente baseado em nucleotídeos e método para tratar um sujeito sofrendo de uma doença requerendo tratamento local com um agente baseado em nucleotídeo.
EP2309980A1 (en) 2008-07-08 2011-04-20 S.I.F.I. Societa' Industria Farmaceutica Italiana Ophthalmic compositions for treating pathologies of the posterior segment of the eye
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
US9255130B2 (en) 2008-07-29 2016-02-09 Academia Sinica Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
JP2012501641A (ja) 2008-09-08 2012-01-26 セレクティス グルタミンシンセターゼ遺伝子からのｄｎａ標的配列を切断するメガヌクレアーゼ変異型及びその使用
DK2334794T3 (en) 2008-09-15 2017-02-20 Children's Medical Center Corp MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
KR101673566B1 (ko) 2008-10-29 2016-11-07 상가모 바이오사이언스 인코포레이티드 글루타민 신테타제 유전자 발현을 불활성화시키기 위한 방법 및 조성물
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US20110023153A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
ES2627552T3 (es) * 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8871905B2 (en) 2009-03-20 2014-10-28 Sangamo Biosciences, Inc. Modification of CXCR4 using engineered zinc finger proteins
EP2427577A4 (en) 2009-05-04 2013-10-23 Hutchinson Fred Cancer Res PSEUDOTYPED RETROVIRAL VECTORS WITH COCAL VESICULOVIRUS SHELL
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
JP2012529287A (ja) 2009-06-11 2012-11-22 トゥールゲン インコーポレイション 部位−特異的ヌクレアーゼを用いた標的ゲノムの再配列
CA2767129C (en) 2009-07-01 2015-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
JP5766188B2 (ja) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド 固形腫瘍に治療剤を送達するための脂質製剤
AU2010275432A1 (en) 2009-07-24 2012-02-02 Sigma-Aldrich Co. Llc. Method for genome editing
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
WO2011028929A2 (en) 2009-09-03 2011-03-10 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP2504439B1 (en) 2009-11-27 2016-03-02 BASF Plant Science Company GmbH Optimized endonucleases and uses thereof
AU2010334799A1 (en) 2009-12-23 2012-07-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Influenza targets
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
TWI688395B (zh) * 2010-03-23 2020-03-21 英翠克頌公司 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途
CN102939377B (zh) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 使用锌指核酸酶对Rosa位点进行基因组编辑
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2569425B1 (en) 2010-05-10 2016-07-06 The Regents of The University of California Endoribonuclease compositions and methods of use thereof
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
US20130183282A1 (en) 2010-05-12 2013-07-18 Cellectis Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof
US8372951B2 (en) 2010-05-14 2013-02-12 National Tsing Hua University Cell penetrating peptides for intracellular delivery
EP2571512B1 (en) * 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
EP2575894B1 (en) 2010-05-28 2015-02-25 Oxford Biomedica (UK) Ltd Delivery of lentiviral vectors to the brain
WO2012012738A1 (en) 2010-07-23 2012-01-26 Sigma-Aldrich Co., Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
US9081737B2 (en) 2010-08-02 2015-07-14 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology POLY (BETA AMINO ALCOHOLS), THEIR PREPARATION AND USES THEREOF
US9549901B2 (en) 2010-09-03 2017-01-24 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
SMT202400520T1 (it) 2011-04-22 2025-01-14 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
PT2702160T (pt) 2011-04-27 2020-07-30 Amyris Inc Métodos para modificação genómica
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
US20140113376A1 (en) * 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
HK1200491A1 (en) 2011-10-06 2015-08-07 Sangamo Biosciences, Inc. Methods and compositions for regulating hiv infection
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
EP2591770B1 (en) 2011-11-14 2016-03-16 Silenseed Ltd Compositions for siRNA delivery and methods of manufacturing and using same
EP2780376B1 (en) 2011-11-18 2017-03-08 Université Laval Methods and products for increasing frataxin levels and uses thereof
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
SG11201405103SA (en) 2012-02-24 2014-09-26 Hutchinson Fred Cancer Res Compositions and methods for the treatment of hemoglobinopathies
HK1203566A1 (zh) 2012-02-29 2015-10-30 桑格摩生物科学股份有限公司 治療亨廷頓氏病的方法和組合物
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
AU2013267209B2 (en) 2012-06-01 2017-02-02 Baruch S. Blumberg Institute Modulation of hepatitis B virus cccDNA transcription
US8614194B1 (en) 2012-07-25 2013-12-24 Kaohsiung Medical University Anionic cell penetrating peptide and its use for intracellular delivery
KR20230065381A (ko) * 2012-07-25 2023-05-11 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
ES2824024T3 (es) 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
WO2014065596A1 (en) 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
AU2013355214B2 (en) * 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
CN119752887A (zh) 2012-12-12 2025-04-04 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
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
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
DK2921557T3 (en) 2012-12-12 2016-11-07 Broad Inst Inc Design of systems, methods and optimized sequence manipulation guide compositions
EP4299741A3 (en) * 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CA2894668A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4481048A3 (en) 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
CN104995302B (zh) 2013-01-16 2021-08-31 爱默蕾大学 Cas9-核酸复合物及其相关用途
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
US9163837B2 (en) 2013-02-27 2015-10-20 Siemens Aktiengesellschaft Flow conditioner in a combustor of a gas turbine engine
WO2014150624A1 (en) 2013-03-14 2014-09-25 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
KR102210319B1 (ko) 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
KR102109069B1 (ko) 2013-03-27 2020-05-12 윌코아게 디바이스들을 인라인 검사 및/또는 테스트하는 방법 및 그와 같은 방법을 수행하는 장치
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US20160122774A1 (en) * 2013-05-29 2016-05-05 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
MY177814A (en) 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
KR102733409B1 (ko) 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
DK3011029T3 (da) * 2013-06-17 2020-03-16 Broad Inst Inc Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering
KR20160056869A (ko) 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3011031B1 (en) * 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
ES2767318T3 (es) * 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
CN106102781A (zh) 2013-08-29 2016-11-09 英联邦高等教育系统天普大学 用于hiv感染的rna指导的治疗的方法和组合物
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
EP3760719A1 (en) 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
KR20160097327A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 ｃａｓ 효소의 발현의 변경을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 방법
CN106103705A (zh) 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
KR20250068794A (ko) * 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
WO2015089419A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
CN103668472B (zh) 2013-12-31 2014-12-24 北京大学 利用CRISPR/Cas9系统构建真核基因敲除文库的方法
WO2015113063A1 (en) 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
JP6323228B2 (ja) 2014-07-18 2018-05-16 富士電機株式会社 電力変換装置
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
WO2016028682A1 (en) * 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
US9944912B2 (en) 2015-03-03 2018-04-17 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
JP6186470B2 (ja) 2016-04-20 2017-08-23 パイオニア株式会社 音響装置、音量制御方法、音量制御プログラム及び記録媒体

Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12473573B2 (en) 2013-09-06 2025-11-18 President And Fellows Of Harvard College Switchable Cas9 nucleases 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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
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)
US11932884B2 (en) 2017-08-30 2024-03-19 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US11643652B2 (en) 2019-03-19 2023-05-09 The Broad Institute, Inc. Methods and compositions for prime editing nucleotide sequences
US12509680B2 (en) 2023-05-31 2025-12-30 The Broad Institute, Inc. Methods and compositions for prime editing nucleotide sequences

Also Published As

Publication number Publication date
MX374532B (es) 2025-03-06
BR122021009076B1 (pt) 2024-02-15
MX2015017313A (es) 2016-11-25
CN105793425A (zh) 2016-07-20
EP3011034A1 (en) 2016-04-27
AU2014281031A1 (en) 2016-01-21
EP3597755A1 (en) 2020-01-22
EP3011034B1 (en) 2019-08-07
IL243195A0 (en) 2016-03-31
AU2014281031B2 (en) 2020-05-21
SG11201510286QA (en) 2016-01-28
JP2016521995A (ja) 2016-07-28
RU2716421C2 (ru) 2020-03-11
CA2915795C (en) 2021-07-13
CN114015726B (zh) 2025-06-24
ZA201508961B (en) 2025-08-27
KR20250012194A (ko) 2025-01-23
WO2014204729A1 (en) 2014-12-24
US20210361779A1 (en) 2021-11-25
CA2915795A1 (en) 2014-12-24
IL243195B (en) 2020-06-30
JP6702858B2 (ja) 2020-06-03
KR20160056869A (ko) 2016-05-20
RU2016101198A (ru) 2019-03-27
RU2016101198A3 (enExample) 2019-03-27
US20160175462A1 (en) 2016-06-23
BR112015031610A2 (pt) 2017-08-22
CN114015726A (zh) 2022-02-08
CN105793425B (zh) 2021-10-26
US10946108B2 (en) 2021-03-16

Similar Documents

Publication Publication Date Title
IL272423A (en) Administration, use and therapeutic applications of the CRISPR-CAS system and preparations for targeting disorders and diseases by using particulate delivery components
SG10201710487VA (en) Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components
IL246117A0 (en) Administration, use and therapeutic applications of crispr-cas systems and preparations for HBV and viral diseases and disorders
IL289736A (en) Provision, use and therapeutic applications of crispr-cas genetic editing systems and genetic editing preparations
SG10201710486XA (en) Delivery and Use of the Crispr-Cas Systems, Vectors and Compositions for Hepatic Targeting and Therapy
LT3294323T (lt) Adenoasocijuotas virusas, skirtas terapiniam pristatymui į centrinę nervų sistemą
WO2014145159A3 (en) Charge-engineered antibodies or compositions of penetration-enhanced targeting proteins and methods of use
ZA201507918B (en) Nasal administration
PH12016500862B1 (en) Autotaxin inhibitor compounds
EP2968739A4 (en) Infusion systems for the controlled delivery of therapeutic agents
EP3068353A4 (en) Systems and methods for subretinal delivery of therapeutic agents
IL244182A0 (en) Preparations, methods and systems for the treatment of skin disorders
IL257194B (en) Targeted delivery of spray-dried formulations to the lungs
PH12016500863A1 (en) Tetracyclic autotaxin inhibitors
PL3646329T3 (pl) Układ do dostarczania pacjentowi wielu infuzji
IL243995B (en) Sustained release preparations made of hyaluronic acid, and their medical uses
ZA201507373B (en) Inhalable pharmaceutical compositions and the inhaler devices containing them
EP2999461A4 (en) Targeted delivery of drugs to the myometrium
AU2014271207A1 (en) Targeted delivery of drugs to the myometrium
DK2964185T3 (da) Præparater til behandlingen af søvnrelaterede luftvejslidelser
ZA201507706B (en) Inhalable pharmaceutical compositions and the inhaler devices containing them
PT3209294T (pt) Composição farmacêutica para uso no aumento do trofismo da mucosa nasal
GB201511126D0 (en) Spray device for the delivery of therapeutic agents
EP2968011A4 (en) Compositions and methods for delivering therapeutic and imaging agents to the sinuses and middle ear
HUE056121T2 (hu) Kompozíció UV-B terápia hatékonyságának növelésére, eljárás annak elõállítására, és alkalmazása