CO2021000051A2 - Guías modificadas de arn para edición génica - Google Patents

Guías modificadas de arn para edición génica

Info

Publication number
CO2021000051A2
CO2021000051A2 CONC2021/0000051A CO2021000051A CO2021000051A2 CO 2021000051 A2 CO2021000051 A2 CO 2021000051A2 CO 2021000051 A CO2021000051 A CO 2021000051A CO 2021000051 A2 CO2021000051 A2 CO 2021000051A2
Authority
CO
Colombia
Prior art keywords
gene editing
modified rna
guidelines
abstract
vitro
Prior art date
Application number
CONC2021/0000051A
Other languages
English (en)
Inventor
Reynald Michael Lescarbeau
Jessica Lynn Seitzer
Amy Madison Rhoden Smith
Seth Alexander
Duncan Brown
Robert Brown
Adhiraj Lanba
Rebecca Lescarbeau
Rubina Parmar
Matthew Roy
Paige Salerno
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CO2021000051A2 publication Critical patent/CO2021000051A2/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • 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
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

RESUMEN Esta divulgación se relaciona con guías modificadas de ARN que tienen actividad mejorada in vitro e in vivo en métodos de edición génica.
CONC2021/0000051A 2018-06-08 2021-01-06 Guías modificadas de arn para edición génica CO2021000051A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862682820P 2018-06-08 2018-06-08
US201862682838P 2018-06-08 2018-06-08
PCT/US2019/036160 WO2019237069A1 (en) 2018-06-08 2019-06-07 Modified guide rnas for gene editing

Publications (1)

Publication Number Publication Date
CO2021000051A2 true CO2021000051A2 (es) 2021-01-18

Family

ID=68770688

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0000051A CO2021000051A2 (es) 2018-06-08 2021-01-06 Guías modificadas de arn para edición génica

Country Status (15)

Country Link
US (1) US20210087568A1 (es)
EP (1) EP3802828A4 (es)
JP (1) JP2021526804A (es)
KR (1) KR20210029772A (es)
CN (1) CN112567036A (es)
AU (1) AU2019282824A1 (es)
BR (1) BR112020024731A2 (es)
CA (1) CA3102950A1 (es)
CO (1) CO2021000051A2 (es)
IL (1) IL278822A (es)
MX (1) MX2020013293A (es)
PH (1) PH12020552100A1 (es)
SG (1) SG11202011539VA (es)
TW (1) TW202016306A (es)
WO (1) WO2019237069A1 (es)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7458785B2 (ja) 2017-01-23 2024-04-01 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 ヒドロキシステロイド１７－βデヒドロゲナーゼ１３（ＨＳＤ１７Ｂ１３）バリアント及びその使用
US11479802B2 (en) 2017-04-11 2022-10-25 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3585159A1 (en) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Cas-transgenic mouse embryonic stem cells and mice and uses thereof
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.
JP7139419B2 (ja) 2017-09-29 2022-09-20 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 ヒト化ｔｔｒ遺伝子座を含む非ヒト動物および使用方法
US10961583B2 (en) 2017-10-11 2021-03-30 Regeneron Phramaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
WO2020198697A1 (en) * 2019-03-28 2020-10-01 Intellia Therapeutics, Inc. Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
CA3137761A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
EP3796776A1 (en) 2019-06-07 2021-03-31 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
US20220290132A1 (en) * 2019-08-06 2022-09-15 The Penn State Research Foundation Engineered CRISPR/Cas9 Systems for Simultaneous Long-term Regulation of Multiple Targets
CN115176001A (zh) * 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
AU2021396403A1 (en) 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compounds and methods for genome editing involving deamination
CA3205042A1 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
WO2022140586A2 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
CN117098840A (zh) 2021-02-08 2023-11-21 因特利亚治疗公司 用于免疫疗法的自然杀伤细胞受体2b4组合物和方法
CN117042794A (zh) 2021-02-08 2023-11-10 因特利亚治疗公司 用于免疫疗法的t细胞免疫球蛋白和粘蛋白结构域3(tim3)组合物和方法
EP4288088A2 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy
WO2023028471A1 (en) 2021-08-24 2023-03-02 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
WO2023081687A1 (en) * 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
WO2023081200A2 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
WO2023185697A2 (en) * 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
CN114574598B (zh) * 2022-04-02 2023-08-08 中国农业科学院北京畜牧兽医研究所 鉴定或辅助鉴定猪5/6肋眼肌面积的方法
WO2023245141A2 (en) * 2022-06-15 2023-12-21 Beam Therapeutics Inc. Compositions and methods for reducing complement activation
TW202408595A (zh) 2022-06-16 2024-03-01 美商英特利亞醫療公司 用於對細胞進行遺傳修飾之方法及組合物
TW202409271A (zh) 2022-06-16 2024-03-01 美商英特利亞醫療公司 用於減少細胞中之ｍｈｃ １類之組成物及方法
WO2024006955A1 (en) 2022-06-29 2024-01-04 Intellia Therapeutics, Inc. Engineered t cells
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
CN117925623B (zh) * 2024-03-20 2024-06-07 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150376587A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
US10513711B2 (en) * 2014-08-13 2019-12-24 Dupont Us Holding, Llc Genetic targeting in non-conventional yeast using an RNA-guided endonuclease
US10900034B2 (en) * 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
EP3280803B1 (en) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
US11279928B2 (en) * 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
WO2017136794A1 (en) * 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
WO2017173054A1 (en) * 2016-03-30 2017-10-05 Intellia Therapeutics, Inc. Lipid nanoparticle formulations for crispr/cas components

Also Published As

Publication number Publication date
CN112567036A (zh) 2021-03-26
JP2021526804A (ja) 2021-10-11
MX2020013293A (es) 2021-05-12
TW202016306A (zh) 2020-05-01
US20210087568A1 (en) 2021-03-25
PH12020552100A1 (en) 2021-08-02
AU2019282824A1 (en) 2021-01-07
KR20210029772A (ko) 2021-03-16
BR112020024731A2 (pt) 2021-03-23
CA3102950A1 (en) 2019-12-12
SG11202011539VA (en) 2020-12-30
WO2019237069A1 (en) 2019-12-12
EP3802828A4 (en) 2022-10-26
IL278822A (en) 2021-01-31
EP3802828A1 (en) 2021-04-14

Similar Documents

Publication Publication Date Title
CO2021000051A2 (es) Guías modificadas de arn para edición génica
CO2019007258A2 (es) Arn guía modificados
PH12019502724A1 (en) Compositions for modulating c9orf72 expression
CO2018004743A2 (es) Proteínas de unión al antígeno anti-tigit y métodos para usarlas
MX2022003891A (es) Anticuerpos anti-muc16 y sus usos.
MD3209310T2 (ro) Compoziții care conțin tulpini bacteriene
MD3209381T2 (ro) Compoziții care conțin tulpini bacteriene
KR20180084895A (ko) 효소 내재화 조성물 및 방법
CR20140441A (es) Anticuerpos anti-lgr5 e inmunoconjugados
MX2016003636A (es) Animales no humanos que tienen un gen de proteina reguladora de señal humanizado.
CO2019005992A2 (es) Composiciones de arni de serpina1 y sus métodos de uso
CO7151527A2 (es) Anticuerpos anti-cd79 b e inmunoconjugados
CO2022009562A2 (es) Arn guía modificados para edición de genes
CL2023002358A1 (es) Constructos de arni para inhibir la expresión de pnpla3 y sus métodos de uso
CR20140529A (es) Anticuerpos e inmunoconjugados contra ly6e y métodos de uso
CR20150047A (es) Anticuerpos anti-cd22 e inmunoconjugados
UY35582A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
CO6690815A2 (es) Métodos y composiciones para inmunoterapia para enfermedad neural
UY34636A (es) Composiciones tópicas que comprenden fipronilo y permetrina y sus métodos de uso
CL2018001788A1 (es) Composiciones y métodos para evaluar el riesgo de ocurrencia de cáncer.
AR106769A1 (es) Composiciones que comprenden cepas bacterianas
IT201600109735A1 (it) Biomarcatori e metodi per la diagnosi in vitro di sclerosi multipla.
EA202092991A1 (ru) Модифицированные направляющие рнк, которые используются для редактирования генома
TH1901007589A (th) องค์ประกอบและวิธีการสำหรับการทำให้เอนไซม์เคลื่อนเข้าภายใน
AR110225A1 (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO