[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

CL2016003247A1 - Composiciones y métodos para la expresión de arns guía usando el promotor h1 - Google Patents

Composiciones y métodos para la expresión de arns guía usando el promotor h1

Info

Publication number
CL2016003247A1
CL2016003247A1 CL2016003247A CL2016003247A CL2016003247A1 CL 2016003247 A1 CL2016003247 A1 CL 2016003247A1 CL 2016003247 A CL2016003247 A CL 2016003247A CL 2016003247 A CL2016003247 A CL 2016003247A CL 2016003247 A1 CL2016003247 A1 CL 2016003247A1
Authority
CL
Chile
Prior art keywords
expression
promoter
methods
compositions
arng
Prior art date
Application number
CL2016003247A
Other languages
English (en)
Inventor
Donald Zack
Ranga Vinod Jaskula
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 CL2016003247A1 publication Critical patent/CL2016003247A1/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-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 receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • C12N15/8616Special methods for targeting systems
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/062Sensory transducers, e.g. photoreceptors; Sensory neurons, e.g. for hearing, taste, smell, pH, touch, temperature, pain
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/60Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • C12N2510/02Cells for production
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14133Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/20Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C12N2830/205Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

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

Abstract

<p>LA MATERIA OBJETO DE LA PRESENTE DIVULGACIÓN PROPORCIONA COMPOSICIONES Y MÉTODOS PARA LA EXPRESIÓN DE ARNs GUÍAS DE CRISPR UTILIZANDO EL PROMOTOR H1. EN PARTICULAR, SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA EL USO DEL PROMOTOR H1 PARA EXPRESAR UN ARN GUÍA (ARNg) DE CRISPR CON ESPECIFICIDAD ALTERADA DEL NUCLEÓTIDO EN 5', ASÍ COMO EL USO DE LA SECUENCIA PROMOTORA H1 COMO UN PROMOTOR BIDIRECCIONAL PARA EXPRESAR LA NUCLEASA Cas9 Y EL ARNg SIMULTÁNEAMENTE. TAMBIÉN SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA LA EXPRESIÓN Y LA REGULACIÓN DE LA EXPRESIÓN ARNg IN VIVO MEDIANTE EL USO DE RIBOZIMAS DE ARN Y APTAZIMAS REGULABLES.</p>
CL2016003247A 2014-06-16 2016-12-16 Composiciones y métodos para la expresión de arns guía usando el promotor h1 CL2016003247A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462012802P 2014-06-16 2014-06-16

Publications (1)

Publication Number Publication Date
CL2016003247A1 true CL2016003247A1 (es) 2017-11-24

Family

ID=54936029

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016003247A CL2016003247A1 (es) 2014-06-16 2016-12-16 Composiciones y métodos para la expresión de arns guía usando el promotor h1

Country Status (16)

Country Link
US (5) US9907863B2 (es)
EP (2) EP3155101B1 (es)
JP (2) JP6930834B2 (es)
KR (1) KR102649341B1 (es)
CN (2) CN106852157B (es)
AU (2) AU2015277369B2 (es)
BR (1) BR112016029178A2 (es)
CA (1) CA2952697A1 (es)
CL (1) CL2016003247A1 (es)
DK (1) DK3155101T3 (es)
EA (1) EA039693B1 (es)
ES (1) ES2788426T3 (es)
IL (2) IL249584B (es)
MX (2) MX2016016833A (es)
SG (2) SG11201610405RA (es)
WO (1) WO2015195621A1 (es)

Families Citing this family (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
CA2908697C (en) 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
CA2948728A1 (en) 2014-03-10 2015-09-17 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
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP3122880B1 (en) 2014-03-26 2021-05-05 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
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
CN105132451B (zh) * 2015-07-08 2019-07-23 电子科技大学 一种CRISPR/Cas9单一转录单元定向修饰骨架载体及其应用
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3199632A1 (en) 2016-01-26 2017-08-02 ACIB GmbH Temperature-inducible crispr/cas system
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017196768A1 (en) * 2016-05-09 2017-11-16 President And Fellows Of Harvard College Self-targeting guide rnas in crispr system
AU2017268458B2 (en) 2016-05-20 2022-07-21 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide RNAS
WO2017223330A1 (en) * 2016-06-22 2017-12-28 Icahn School Of Medicine At Mount Sinai Viral delivery of rna utilizing self-cleaving ribozymes and crispr-based applications thereof
MX2019000188A (es) * 2016-07-05 2019-06-20 Univ Johns Hopkins Composiciones y metodos basados en crispr/cas9 para el tratamiento del cancer.
AU2017292667A1 (en) * 2016-07-05 2019-02-21 The Johns Hopkins University Compositions and methods comprising improvements of CRISPR guide RNAs using the H1 promoter
CN109890424A (zh) * 2016-07-05 2019-06-14 约翰霍普金斯大学 用于治疗视网膜变性的基于crispr/cas9的组合物和方法
RU2721125C1 (ru) 2016-07-29 2020-05-18 Регенерон Фармасьютикалз, Инк. Мыши, содержащие мутации, вследствие которых экспрессируется укороченный на с-конце фибриллин-1
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20230095129A (ko) 2016-08-03 2023-06-28 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
IL247752A0 (en) 2016-09-11 2016-11-30 Yeda Res & Dev Compositions and methods for modulating gene expression for site-directed mutagenesis
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018130830A1 (en) * 2017-01-11 2018-07-19 Oxford University Innovation Limited Crispr rna
CA3047429A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hsd17b13 variants and uses thereof
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
JP7327803B2 (ja) * 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症(als)を処置する方法
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) * 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
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
WO2019028032A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF
EP3585161A1 (en) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
BR112020001996A2 (pt) 2017-07-31 2020-08-18 Regeneron Pharmaceuticals, Inc. animal não humano, e, métodos para testar e para otimizar a capacidade de uma crispr/cas nuclease de excisar um ácido nucleico genômico in vivo, para testar a recombinação induzida por crispr/cas de um ácido nucleico genômico com um ácido nucleico de doador exógeno in vivo e para otimizar a capacidade de crispr/cas de induzir a recombinação de um ácido nucleico genômico com um ácido nucleico de doador exógeno in vivo.
US20200260698A1 (en) * 2017-08-18 2020-08-20 The Board Of Regents Of The University Of Texas System Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing
EP3676376A2 (en) 2017-08-30 2020-07-08 President and Fellows of Harvard College High efficiency base editors comprising gam
EP3676378A4 (en) * 2017-08-31 2021-06-09 The New York Genome Center PROCEDURES AND COMPOSITIONS USING CRIMP-CPF1 AND PAIRED GUIDE-CRISPR-RNA FOR PROGRAMMABLE GENOMIC DELETIONS
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
NZ761329A (en) 2017-09-29 2023-04-28 Regeneron Pharma Non-human animals comprising a humanized ttr locus and methods of use
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20200354419A1 (en) * 2017-11-03 2020-11-12 Hunterian Medicine Llc Compositions and methods of use thereof for the treatment of duchenne muscular dystrophy
US11834648B2 (en) * 2017-11-06 2023-12-05 Shenzhen Cell Inspire Biotechnology Co., Ltd. Human induced pluripotent stem cell lines for modeling Alzheimer's disease and usage thereof
US10662425B2 (en) 2017-11-21 2020-05-26 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
AU2019236210A1 (en) * 2018-03-14 2020-09-10 Arbor Biotechnologies, Inc. Novel CRISPR DNA targeting enzymes and systems
CA3093702A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2019183123A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
US20210189426A1 (en) * 2018-05-15 2021-06-24 The Children's Hospital Of Philadelphia Crispr interference based htt allelic suppression and treatment of huntington disease
EP3794130A4 (en) 2018-05-16 2022-07-27 Synthego Corporation METHODS AND SYSTEMS FOR DESIGN AND USE OF GUIDE RNA
US11207425B2 (en) * 2018-09-28 2021-12-28 City University Of Hong Kong Guide RNA molecule and method for treating cancer
WO2020086996A1 (en) * 2018-10-26 2020-04-30 North Carolina State University Compositions and methods related to nucleic acid anticoagulants
CN113423831B (zh) 2018-12-20 2023-03-10 瑞泽恩制药公司 核酸酶介导的重复扩增
WO2020176552A1 (en) * 2019-02-25 2020-09-03 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2020191245A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
AU2020256225A1 (en) 2019-04-03 2021-09-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
CN117178959A (zh) 2019-04-04 2023-12-08 瑞泽恩制药公司 包括人源化凝血因子12基因座的非人动物
MX2021012511A (es) * 2019-04-14 2022-01-06 Univ Duke Delecion mediada por vector aav de punto activo mutacional grande para el tratamiento de distrofia muscular de duchenne.
CN110029121A (zh) * 2019-04-17 2019-07-19 遵义医学院附属医院 一种铜绿假单胞菌CRISPRi系统的构建方法
WO2020227307A1 (en) * 2019-05-07 2020-11-12 Refuge Biotechnologies, Inc. Systems and methods for nuclear localization of gene modulating polypeptides
JP2022534867A (ja) 2019-06-04 2022-08-04 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 ベータスリップ変異を有するヒト化ttr遺伝子座を含む非ヒト動物と使用方法
BR112021022722A2 (pt) 2019-06-07 2022-01-04 Regeneron Pharma Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente
WO2020252340A1 (en) 2019-06-14 2020-12-17 Regeneron Pharmaceuticals, Inc. Models of tauopathy
EP4028063A1 (en) 2019-09-13 2022-07-20 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
WO2021092513A1 (en) 2019-11-08 2021-05-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
AU2021205965A1 (en) * 2020-01-10 2022-09-01 Solid Biosciences Inc. Viral vector for combination therapy
EP4096396A1 (en) 2020-01-28 2022-12-07 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized pnpla3 locus and methods of use
US20230081547A1 (en) 2020-02-07 2023-03-16 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus and methods of use
US20230210096A1 (en) * 2020-02-21 2023-07-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Non-human animal models for aging and/or neurodegeneration
EP4114946A1 (en) 2020-03-04 2023-01-11 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
EP3889259A1 (en) 2020-03-30 2021-10-06 IMBA-Institut für Molekulare Biotechnologie GmbH Internal standard for crispr guide rna
KR20230019843A (ko) 2020-05-08 2023-02-09 더 브로드 인스티튜트, 인코퍼레이티드 표적 이중 가닥 뉴클레오티드 서열의 두 가닥의 동시 편집을 위한 방법 및 조성물
EP4256052A1 (en) 2020-12-02 2023-10-11 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
CA3204046A1 (en) * 2021-01-05 2022-07-14 James M. Carothers Modular kinetically-controlled functional rna constructs and related compositions, systems, and methods
WO2022212766A2 (en) * 2021-03-31 2022-10-06 Hunterian Medicine Llc Compact promoters for gene expression
EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
WO2023102225A2 (en) * 2021-12-03 2023-06-08 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
IL314482A (en) 2022-02-02 2024-09-01 Regeneron Pharma Anti-TFR:GAA and anti-CD63:GAA insertions for the treatment of Pompe disease
EP4223877A1 (en) * 2022-02-08 2023-08-09 Eberhard Karls Universität Tübingen Medizinische Fakultät System and method for editing genomic dna to modulate splicing
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
KR20240000814A (ko) * 2022-06-24 2024-01-03 연세대학교 산학협력단 망막색소상피에 특이적으로 작동하는 CRISPR/Cas 복합체를 유효성분으로 포함하는 망막질환 치료용 약학조성물
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
WO2024098002A1 (en) 2022-11-04 2024-05-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
US20240173426A1 (en) 2022-11-14 2024-05-30 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024107836A1 (en) * 2022-11-15 2024-05-23 The Johns Hopkins University Methods for intron-driven gene editing and control
CN117384906B (zh) * 2023-12-08 2024-03-12 中国农业大学三亚研究院 一种来源于aanat基因的启动子元件及其应用

Family Cites Families (48)

* 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
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
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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
JPS61257185A (ja) * 1985-05-08 1986-11-14 Mitsubishi Chem Ind Ltd 新規なプロモ−タ
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
ATE141646T1 (de) 1986-04-09 1996-09-15 Genzyme Corp Genetisch transformierte tiere, die ein gewünschtes protein in milch absondern
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
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
US5270163A (en) 1990-06-11 1993-12-14 University Research Corporation Methods for identifying nucleic acid ligands
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5582981A (en) 1991-08-14 1996-12-10 Gilead Sciences, Inc. Method for identifying an oligonucleotide aptamer specific for a target
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
US5756291A (en) 1992-08-21 1998-05-26 Gilead Sciences, Inc. Aptamers specific for biomolecules and methods of making
WO1997042317A1 (en) 1996-05-07 1997-11-13 T Cell Sciences, Inc. APTAMERS FOR COMPLEMENT PROTEIN C3b
AU1252099A (en) 1997-11-26 1999-06-15 Medical Research Council Improved selex procedure and an anti-cd4 aptamer
US6180348B1 (en) 1998-04-20 2001-01-30 Weihua Li Method of isolating target specific oligonucleotide ligands
AU1201600A (en) 1998-10-08 2000-04-26 University Of Massachusetts Controlling gene expression in living cells
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
EP1287362A4 (en) * 2000-05-25 2004-05-12 Univ Virginia COMPOSITIONS AND METHODS FOR MODULATING TUMOR SPECIFIC EXPRESSION
KR20050026384A (ko) * 2002-04-26 2005-03-15 내셔날 인스티튜트 오브 어드밴스드 인더스트리얼 사이언스 앤드 테크놀로지 RNAi효과를 가지는 스템 루프 형 RNA 분자 발현 시스템
US20060171924A1 (en) * 2003-10-03 2006-08-03 Ke Luo Bidirectional promoters for small RNA expression
US9315862B2 (en) 2004-10-05 2016-04-19 California Institute Of Technology Aptamer regulated nucleic acids and uses thereof
JP2008532519A (ja) * 2005-03-09 2008-08-21 ボード オブ リージェンツ, ザ ユニバーシティ オブ テキサス システム 遺伝子発現のインビボ画像化に関する方法および組成物
US8158595B2 (en) * 2006-11-09 2012-04-17 California Institute Of Technology Modular aptamer-regulated ribozymes
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
CN101544976B (zh) * 2009-05-06 2011-07-20 北京林业大学 双向启动子双可视荧光蛋白报告基因植物表达载体
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
KR20140066193A (ko) * 2011-09-01 2014-05-30 힙 이노베이션 테크놀러지 엘엘씨 라이너가 설치된 대퇴골 컵
US20160017366A1 (en) * 2012-12-06 2016-01-21 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2997146A4 (en) * 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
EP4385567A3 (en) 2013-08-29 2024-08-14 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
CN103614415A (zh) * 2013-11-27 2014-03-05 苏州同善生物科技有限公司 一种基于crispr基因敲除技术建立肥胖症大鼠动物模型的方法
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
CN108350446A (zh) 2015-07-02 2018-07-31 约翰霍普金斯大学 基于crispr/cas9的治疗
US10393912B2 (en) 2015-07-02 2019-08-27 Weatherford Technology Holdings, Llc Method of and apparatus for inverting three-dimensional fluid property distribution over the (T1,T2,D)domain from NMR measurements
MX2019000188A (es) * 2016-07-05 2019-06-20 Univ Johns Hopkins Composiciones y metodos basados en crispr/cas9 para el tratamiento del cancer.
CN109890424A (zh) * 2016-07-05 2019-06-14 约翰霍普金斯大学 用于治疗视网膜变性的基于crispr/cas9的组合物和方法

Also Published As

Publication number Publication date
SG10202002486QA (en) 2020-04-29
JP2017519500A (ja) 2017-07-20
EP3674408A1 (en) 2020-07-01
MX2020007078A (es) 2020-09-09
US20190365928A1 (en) 2019-12-05
AU2015277369B2 (en) 2021-08-19
BR112016029178A2 (pt) 2017-10-17
CN114686483A (zh) 2022-07-01
EP3155101A1 (en) 2017-04-19
AU2015277369A1 (en) 2017-02-02
EP3155101B1 (en) 2020-01-29
US10369234B2 (en) 2019-08-06
US9907863B2 (en) 2018-03-06
US20200268907A1 (en) 2020-08-27
SG11201610405RA (en) 2017-01-27
MX2016016833A (es) 2017-10-04
EP3155101A4 (en) 2018-04-04
CN106852157B (zh) 2022-04-12
AU2021269446A1 (en) 2021-12-16
JP6930834B2 (ja) 2021-09-01
US20180369420A1 (en) 2018-12-27
EA201692510A1 (ru) 2017-06-30
US10668173B2 (en) 2020-06-02
DK3155101T3 (da) 2020-05-04
IL249584A0 (en) 2017-02-28
EA039693B1 (ru) 2022-02-28
KR20170020867A (ko) 2017-02-24
WO2015195621A1 (en) 2015-12-23
KR102649341B1 (ko) 2024-03-18
CA2952697A1 (en) 2015-12-23
CN106852157A (zh) 2017-06-13
US10406245B2 (en) 2019-09-10
ES2788426T3 (es) 2020-10-21
JP2021052809A (ja) 2021-04-08
US20160074535A1 (en) 2016-03-17
US11896679B2 (en) 2024-02-13
IL290576A (en) 2022-04-01
IL249584B (en) 2022-04-01
US20180200388A1 (en) 2018-07-19

Similar Documents

Publication Publication Date Title
CL2016003247A1 (es) Composiciones y métodos para la expresión de arns guía usando el promotor h1
PH12017502255A1 (en) Nrf2 regulators
BR112019011450A2 (pt) células naturais killer modificadas e uso das mesmas
SG10201809290SA (en) Compositions and Methods to Treating Hemoglobinopathies
MX2018006801A (es) Ensamble de contenedor.
AU366476S (en) Bottle
MX2018005132A (es) Agonistas del receptor de glucagon.
CO2017004728A2 (es) Composiciones y métodos para inhibir la expresión del gen hao1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa)
PH12016501151A1 (en) Nrf2 regulators
SV2015005107A (es) Composiciones y métodos para activar la señalización que depende del estimulador del gen de interferon
BR112017009374A2 (pt) ?composições de ramnolipídeo de baixa viscosidade concentradas?
EP4273238A3 (en) Compositions comprising polypeptides having xylanase activity and polypeptides having arabinofuranosidase activity
CL2017001336A1 (es) Nuevos aminoácidos ester fosforamidatos 2&#39; y/o 5&#39;, 3&#39; deoxiadenosina derivativos como compuestos anticancerígenos
PE20170088A1 (es) Anticuerpos especificos del receptor del factor 1 de crecimiento similar a la insulina y usos de los mismos
CL2017001100A1 (es) Nuevas triazolo[4,5-d]pirimidinas
PH12016500683B1 (en) Synthetic route to 2`-deoxy-2`,2`-difluorotetrahydrouridines
UY35947A (es) Compuestos antagonistas selectivos de n-metil-d-aspartato (nmda) subtipo nr2b y composiciones que los contienen
UY35946A (es) Composiciones que contienen (r)-3-((3s,4s)-3-fluoro-4-(4-hidroxifenil)piperidin-1-il)-1-(4-metilbencil)pirrolidin-2-ona y sus profármacos, como ligandos para el receptor de nmda nr2b
PH12018501844A1 (en) Liraglutide in cardiovascular conditions
PH12017501601A1 (en) Use of isolated fractions of mastic gum for treating optic neuropathy
UY35783A (es) Elementos regulatorios de zea mays y usos de los mismos.
NZ741423A (en) Bio-based n-acetyl-l-methionine and use thereof
DK201670798A1 (en) Novel spinach and method for producing the same
SA516370731B1 (ar) عملية لتحضير جزء وجبة تباع الشمس عالية البروتين
HK1254516A1 (zh) 包含3-o-棓酰-3,3&#39;,5,5&#39;,7-五羥基黃烷的保濕用組合物