[go: up one dir, main page]

MX2019000262A - Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas. - Google Patents

Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas.

Info

Publication number
MX2019000262A
MX2019000262A MX2019000262A MX2019000262A MX2019000262A MX 2019000262 A MX2019000262 A MX 2019000262A MX 2019000262 A MX2019000262 A MX 2019000262A MX 2019000262 A MX2019000262 A MX 2019000262A MX 2019000262 A MX2019000262 A MX 2019000262A
Authority
MX
Mexico
Prior art keywords
methods
crispr
cas9
based compositions
treating retinal
Prior art date
Application number
MX2019000262A
Other languages
English (en)
Inventor
Welsbie Derek
Zack Donald
Jaskula-Ranga Vinod
Bunz Fred
Original Assignee
Univ Johns Hopkins
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 Univ Johns Hopkins filed Critical Univ Johns Hopkins
Publication of MX2019000262A publication Critical patent/MX2019000262A/es

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/11—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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • 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)
    • 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
    • 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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—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
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional

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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Prostheses (AREA)

Abstract

En este documento se describen métodos para tratar una degeneración retiniana en un sujeto, tal como la amaurosis congénita de Leber (LCA), la retinitis pigmentosa (RP) y el glaucoma. En el presente documento también se proporcionan métodos para alterar la expresión de uno o más productos genéticos en una célula, como una célula ganglionar retiniana. Dichos métodos pueden comprender la utilización de un sistema de nucleasa modificado, como el sistema de repeticiones palindrómicas cortas agrupadas y regularmente interespaciadas (CRISPR) que comprende un promotor H1 bidireccional y RNAg dirigidos a genes relacionados con la degeneración retiniana empaquetados en una única partícula de virus adenoasociado (AAV) compacta.
MX2019000262A 2016-07-05 2017-07-05 Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas. MX2019000262A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662358337P 2016-07-05 2016-07-05
PCT/US2017/040745 WO2018009562A1 (en) 2016-07-05 2017-07-05 Crispr/cas9-based compositions and methods for treating retinal degenerations

Publications (1)

Publication Number Publication Date
MX2019000262A true MX2019000262A (es) 2019-05-27

Family

ID=60913143

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019000262A MX2019000262A (es) 2016-07-05 2017-07-05 Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas.

Country Status (14)

Country Link
US (2) US20200080108A1 (es)
EP (1) EP3481434A4 (es)
JP (1) JP2019520391A (es)
KR (1) KR20190039703A (es)
CN (1) CN109890424A (es)
AU (1) AU2017293773A1 (es)
BR (1) BR112019000057A2 (es)
CA (1) CA3029874A1 (es)
CL (1) CL2019000024A1 (es)
EA (1) EA201990212A1 (es)
IL (1) IL264028A (es)
MX (1) MX2019000262A (es)
SG (2) SG10202109385QA (es)
WO (1) WO2018009562A1 (es)

Families Citing this family (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015195621A1 (en) * 2014-06-16 2015-12-23 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas using the h1 promoter
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOM ENGINEERING WITH TYPE I CRISPR SYSTEMS IN EUKARYOTIC CELLS
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US12214056B2 (en) 2016-07-19 2025-02-04 Duke University Therapeutic applications of CPF1-based genome editing
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
IL308426B1 (en) 2016-08-03 2025-11-01 Harvard College Adenosine nucleobase editors and uses thereof
JP7201153B2 (ja) 2016-08-09 2023-01-10 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 プログラム可能ｃａｓ９－リコンビナーゼ融合タンパク質およびその使用
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20190153440A1 (en) 2017-11-21 2019-05-23 Casebia Therapeutics Llp Materials and methods for treatment of autosomal dominant retinitis pigmentosa
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
US12331328B2 (en) 2018-03-23 2025-06-17 Massachusetts Eye And Ear Infirmary CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated usher syndrome
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
AU2019314208A1 (en) * 2018-08-02 2021-02-18 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
DE112019005166B4 (de) 2018-10-16 2024-12-24 BlueAllele Corporation (n.d.Ges.d. Staates Delaware) Dna-polynukleotid zur gezielten insertion von dna in gene
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN113166769A (zh) * 2018-11-01 2021-07-23 蓝色等位基因有限责任公司 用于改变遗传病症的基因表达的方法
EP3911735A4 (en) * 2019-01-16 2023-07-12 Beam Therapeutics, Inc. MODIFIED IMMUNE CELLS WITH INCREASED ANTINEOPLASTIC ACTIVITY AND RESISTANCE TO IMMUNOSUPPRESSION
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3924470A4 (en) * 2019-02-15 2022-12-14 Exhaura, Ltd. DUAL LEUCINE ZIPPER KINASE INHIBITORS FOR GENE THERAPY
EP3931326A1 (en) * 2019-02-25 2022-01-05 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
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
JP7609436B2 (ja) * 2019-07-12 2025-01-07 国立研究開発法人理化学研究所 顕性型変異遺伝子に由来する疾患の治療剤
WO2021046155A1 (en) * 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021055855A1 (en) * 2019-09-20 2021-03-25 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4069845A1 (en) 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
US20230054569A1 (en) 2019-12-18 2023-02-23 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
EP4121443A4 (en) * 2020-03-20 2024-05-15 Dignity Health METHOD FOR THE PRODUCTION AND ISOLATION OF ADENO-ASSOCIATED VIRUS
IT202000008014A1 (it) 2020-04-15 2021-10-15 Fond Telethon RNA guida e loro usi
US20230149439A1 (en) * 2020-04-21 2023-05-18 Chigenovo Co., Ltd. Rho-adrp gene editing-based methods and compositions
WO2021212686A1 (zh) * 2020-04-21 2021-10-28 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
EP4146804A1 (en) 2020-05-08 2023-03-15 The Broad Institute Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111850044A (zh) * 2020-07-16 2020-10-30 中国科学技术大学 基于在体基因敲除的视网膜色素变性猕猴模型构建方法
US20230392132A1 (en) * 2020-10-21 2023-12-07 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
WO2023285431A1 (en) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions and methods for allele specific treatment of retinitis pigmentosa
KR20230016751A (ko) * 2021-07-26 2023-02-03 서울대학교산학협력단 염기 편집기 및 이의 용도
EP4399307A4 (en) * 2021-09-08 2026-01-21 Flagship Pioneering Innovations Vi Llc HBB COMPOSITIONS AND MODULATION METHODS
CN114550817B (zh) * 2022-01-25 2022-12-23 云南大学 基于多特征的ctcf介导染色质环预测方法
EP4223877A1 (en) 2022-02-08 2023-08-09 Eberhard Karls Universität Tübingen Medizinische Fakultät System and method for editing genomic dna to modulate splicing
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
IL320664A (en) * 2022-11-02 2025-07-01 Emendobio Inc Compositions and methods for transcription factor 4 (tcf4) repeat expansion excision
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
EP4442826A1 (en) * 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
CN117887841B (zh) * 2024-03-08 2024-07-26 北京市眼科研究所 基于CRISPR/Cas12的一步法检测Leber遗传性视神经病变的组合物、试剂盒及应用
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006118308A1 (ja) * 2005-05-02 2006-11-09 Toray Industries, Inc. 食道ガン及び食道ガン転移診断のための組成物及び方法
WO2013134766A2 (en) * 2012-03-09 2013-09-12 The Johns Hopkins University Identification of molecular pathways and methods of use thereof for treating retinal neurodegeneration and other neurodegenerative disorders
EP2852388A4 (en) * 2012-05-23 2016-01-13 Univ Johns Hopkins COMPOUNDS AND METHOD FOR USE THEREOF FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
CN116622704A (zh) * 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
CA2932478A1 (en) * 2013-12-12 2015-06-18 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
US9938521B2 (en) * 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
PE20161252A1 (es) * 2014-03-21 2016-11-30 Genzyme Corp Terapia genica para la retinitis pigmentaria
US12460231B2 (en) * 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
WO2015195621A1 (en) * 2014-06-16 2015-12-23 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas using the h1 promoter
WO2016034680A1 (en) * 2014-09-05 2016-03-10 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
DK3289080T3 (da) * 2015-04-30 2021-11-08 Univ Columbia Genterapi til autosomalt dominante sygdomme

Also Published As

Publication number Publication date
US20240336934A1 (en) 2024-10-10
SG11201900049QA (en) 2019-02-27
KR20190039703A (ko) 2019-04-15
JP2019520391A (ja) 2019-07-18
EP3481434A4 (en) 2020-06-24
CL2019000024A1 (es) 2019-06-21
IL264028A (en) 2019-02-03
US20200080108A1 (en) 2020-03-12
EA201990212A1 (ru) 2020-09-07
CA3029874A1 (en) 2018-01-11
WO2018009562A1 (en) 2018-01-11
BR112019000057A2 (pt) 2019-04-02
SG10202109385QA (en) 2021-10-28
EP3481434A1 (en) 2019-05-15
CN109890424A (zh) 2019-06-14
AU2017293773A1 (en) 2019-02-21

Similar Documents

Publication Publication Date Title
MX2019000262A (es) Composiciones basadas en crispr/cas9 y metodos para el tratamiento de degeneraciones retinianas.
EA201990214A1 (ru) Композиции на основе crispr/cas9 и способы лечения рака
WO2014197748A3 (en) Rna-guided gene editing and gene regulation
WO2019006418A3 (en) ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2017075477A8 (en) Compositions and methods for activating "stimulator of interferon gene"-dependent signalling
PH12017501506A1 (en) Compositions and methods for activating``stimulator of interferon gene`` -dependent signalling
WO2017181107A3 (en) Modified cpf1 mrna, modified guide rna, and uses thereof
MX2017016921A (es) Tratamientos basados en crispr / cas9.
WO2017066497A3 (en) Genome engineering with type i crispr systems in eukaryotic cells
WO2015138510A8 (en) Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
IL290678B2 (en) Novel crispr enzymes and systems
MY194058A (en) Cyclic dinucleotides for treating conditions associated with sting activity such as cancer
EP4495133A3 (en) Methods of isolating t cells having antigenic specificity for a p53 cancer-specific mutation
ZA201607053B (en) Defined composition gene modified t-cell products
MY175308A (en) Compositions and methods for activating "stimulator of interferon gene"-dependent signalling
SG10201907399RA (en) Enhanced delivery of viral particles to the striatum and cortex
NZ730803A (en) Methods for the preparation of ribosides
EP2552483A4 (en) USE OF ANTIBODIES TO REDUCE THE NUMBER OF CELLS CONTAINING ANTIGENS AND DENDRITIC CELLS
AU2021216418A8 (en) Compositions and methods for targeting, editing or modifying human genes
MX2022010792A (es) Sistemas y metodos para refinar licores.
MX2024001055A (es) Composiciones de vectores de acidos nucleicos.
AU2017256910A1 (en) Optogenetic visual restoration using Chrimson
WO2012064146A3 (ko) Ｇｋｎ １을 함유하는 항암용 조성물
MY204271A (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
EP4062920A4 (en) MEDICINE FOR THE TREATMENT OF CANCER, COMBINATION MEDICINE, MEDICINAL COMPOSITION, IMMUNOREACTIVE CELL, NUCLEIC ACID DELIVERY VEHICLE AND PRODUCT