KR102553518B1 - Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물 - Google Patents

Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물 Download PDF

Info

Publication number
KR102553518B1
KR102553518B1 KR1020177037584A KR20177037584A KR102553518B1 KR 102553518 B1 KR102553518 B1 KR 102553518B1 KR 1020177037584 A KR1020177037584 A KR 1020177037584A KR 20177037584 A KR20177037584 A KR 20177037584A KR 102553518 B1 KR102553518 B1 KR 102553518B1
Authority
KR
South Korea
Prior art keywords
ltr
nucleic acid
hiv
sequence
grna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
KR1020177037584A
Other languages
English (en)
Korean (ko)
Other versions
KR20180023911A (ko
Inventor
카멜 칼리리
웬후이 휴
영강 장
Original Assignee
템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션
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 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 filed Critical 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션
Priority to KR1020237022586A priority Critical patent/KR20230110368A/ko
Publication of KR20180023911A publication Critical patent/KR20180023911A/ko
Application granted granted Critical
Publication of KR102553518B1 publication Critical patent/KR102553518B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • 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
    • 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
    • C12N15/1131—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 viruses
    • C12N15/1132—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 viruses against retroviridae, e.g. HIV
    • 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)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • AIDS & HIV (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020177037584A 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물 Active KR102553518B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020237022586A KR20230110368A (ko) 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562169384P 2015-06-01 2015-06-01
US62/169,384 2015-06-01
US201562169633P 2015-06-02 2015-06-02
US62/169,633 2015-06-02
US201662308320P 2016-03-15 2016-03-15
US62/308,320 2016-03-15
PCT/US2016/035141 WO2016196539A2 (en) 2015-06-01 2016-06-01 Methods and compositions for rna-guided treatment of hiv infection

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020237022586A Division KR20230110368A (ko) 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물

Publications (2)

Publication Number Publication Date
KR20180023911A KR20180023911A (ko) 2018-03-07
KR102553518B1 true KR102553518B1 (ko) 2023-07-07

Family

ID=57442165

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020177037584A Active KR102553518B1 (ko) 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
KR1020237022586A Withdrawn KR20230110368A (ko) 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020237022586A Withdrawn KR20230110368A (ko) 2015-06-01 2016-06-01 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물

Country Status (15)

Country Link
US (3) US11298410B2 (enExample)
EP (2) EP3919622A1 (enExample)
JP (1) JP6968416B2 (enExample)
KR (2) KR102553518B1 (enExample)
CN (1) CN108025188A (enExample)
AU (3) AU2016270702B2 (enExample)
CA (1) CA2987927C (enExample)
DK (1) DK3302709T3 (enExample)
EA (1) EA037359B1 (enExample)
ES (1) ES2886480T3 (enExample)
MX (1) MX2017015582A (enExample)
PL (1) PL3302709T3 (enExample)
SG (1) SG10201914137YA (enExample)
WO (1) WO2016196539A2 (enExample)
ZA (1) ZA201708236B (enExample)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
AU2014312123A1 (en) 2013-08-29 2016-03-17 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase 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
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
KR102553518B1 (ko) 2015-06-01 2023-07-07 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
JP2018527943A (ja) * 2015-09-28 2018-09-27 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション Ｒｎａ誘導性の、ｈｉｖ感染の処置のための、方法および組成物
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
KR20250103795A (ko) 2016-08-03 2025-07-07 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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of 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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
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
WO2018194876A1 (en) * 2017-04-17 2018-10-25 Temple University- Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
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
US12415000B2 (en) * 2017-07-07 2025-09-16 The Broad Institute, Inc. CRISPR system based antiviral therapy
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CN111373041B (zh) * 2017-09-26 2024-10-11 伊利诺伊大学理事会 用于基因组编辑和调节转录的crispr/cas系统和方法
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020150499A1 (en) * 2019-01-16 2020-07-23 Fred Hutchinson Cancer Research Center Methods to tag and isolate cells infected with the human immunodeficiency virus
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020369599A1 (en) * 2019-10-23 2022-05-12 Monsanto Technology Llc Compositions and methods for RNA-templated editing in plants
WO2021178924A1 (en) * 2020-03-05 2021-09-10 Board Of Regents Of The University Of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
MX2023000661A (es) * 2020-07-13 2023-07-03 Alexandra L Howell Métodos y composiciones para la eficiencia y especificidad de arn guía de crispr/cas9 frente a aislados de vih-1 genéticamente diferentes.
CN112322587A (zh) * 2020-09-17 2021-02-05 杭州市第一人民医院 一种带Cas9基因的人源可诱导多能干细胞系、构建方法、鉴定方法及应用
US20240165266A1 (en) * 2021-03-17 2024-05-23 Board Of Regents Of The University Of Nebraska Lipid nanoparticle formulations and methods of use thereof
WO2024103049A1 (en) * 2022-11-11 2024-05-16 The United States Government As Represented By The Department Of Veterans Affairs Cd33 specific exosomes and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US20160040165A1 (en) 2013-04-04 2016-02-11 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for in vivo excision of hiv-1 proviral dna

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
RU2690935C2 (ru) 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CA2917638C (en) 2013-07-09 2024-09-10 Harvard College RNA MULTIPLEX GUIDED GENOMIC ENGINEERING
SG10201800213VA (en) 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CN103923911B (zh) * 2014-04-14 2016-06-08 上海金卫生物技术有限公司 CRISPR-Cas9特异性敲除人CCR5基因的方法以及用于特异性靶向CCR5基因的sgRNA
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CN104480144B (zh) * 2014-12-12 2017-04-12 武汉大学 用于艾滋病基因治疗的CRISPR/Cas9重组慢病毒载体及其慢病毒
MA41349A (fr) 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
CN104726449A (zh) 2015-03-23 2015-06-24 国家纳米科学中心 一种用于预防和/或治疗HIV的CRISPR-Cas9系统及其制备方法和用途
KR102553518B1 (ko) * 2015-06-01 2023-07-07 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
JP2018527943A (ja) 2015-09-28 2018-09-27 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション Ｒｎａ誘導性の、ｈｉｖ感染の処置のための、方法および組成物
CN108778343A (zh) 2015-10-16 2018-11-09 天普大学-联邦高等教育系统 利用cpf1进行rna向导的基因编辑的方法和组合物
AU2016341919A1 (en) 2015-10-21 2018-04-19 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating hepatitis b virus
RU2018124657A (ru) 2015-12-09 2020-01-09 Эксижн Биотерапевтикс, Инк. Способы редактирования генов и композиции для устранения риска активации вируса jc и пмл (прогрессирующая мультифокальная лейкоэнцефалопатия) во время иммуносупрессивной терапии
JP2019506156A (ja) 2016-01-25 2019-03-07 エクシジョン バイオセラピューティクス インコーポレイテッド Ｈｉｖ感染症のｒｎａ誘導型治療のための方法及び組成物
EP3417062B1 (en) 2016-02-15 2024-06-26 Temple University - Of The Commonwealth System of Higher Education Excision of retroviral nucleic acid sequences
US9925425B2 (en) 2016-03-17 2018-03-27 Acushnet Company Golf ball incorporating at least three adjacent ionomeric and/or HNP-based layers having multiple related property gradients there between
WO2017176529A1 (en) 2016-04-06 2017-10-12 Temple Univesity-Of The Commonwealth System Of Higher Education Compositions for eradicating flavivirus infections in subjects
WO2017192172A1 (en) 2016-05-05 2017-11-09 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
US20190256844A1 (en) 2016-06-07 2019-08-22 Temple University - Of The Commonwealth System Of Higher Education Rna guided compositions for preventing and treating hepatitis b virus infections
US20190085326A1 (en) 2016-09-14 2019-03-21 Temple University - Of The Commonwealth System Of Higher Education Negative feedback regulation of HIV-1 by gene editing strategy
CN110023494A (zh) 2016-09-30 2019-07-16 加利福尼亚大学董事会 Rna指导的核酸修饰酶及其使用方法
US20190365862A1 (en) 2016-10-12 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Combination therapies for eradicating flavivirus infections in subjects
US11299430B2 (en) 2016-11-30 2022-04-12 Hrl Laboratories, Llc Formulations with active functional additives for 3D printing of preceramic polymers, and methods of 3D-printing the formulations
CN110431230A (zh) 2017-01-26 2019-11-08 切除生物治疗公司 慢病毒和非整合型慢病毒作为病毒载体实施crispr治疗
WO2018152418A1 (en) 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
WO2018194876A1 (en) 2017-04-17 2018-10-25 Temple University- Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160040165A1 (en) 2013-04-04 2016-02-11 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for in vivo excision of hiv-1 proviral dna
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US20160017301A1 (en) 2013-08-29 2016-01-21 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection

Also Published As

Publication number Publication date
CA2987927C (en) 2024-03-19
US12251429B2 (en) 2025-03-18
KR20180023911A (ko) 2018-03-07
SG10201914137YA (en) 2020-02-27
AU2025204187A1 (en) 2025-06-26
ES2886480T3 (es) 2021-12-20
CA2987927A1 (en) 2016-12-08
HK1253882A1 (en) 2019-07-05
ZA201708236B (en) 2019-12-18
US20200282025A1 (en) 2020-09-10
US20210052709A1 (en) 2021-02-25
MX2017015582A (es) 2018-09-06
US11273209B2 (en) 2022-03-15
AU2022201875A1 (en) 2022-04-07
EA037359B1 (ru) 2021-03-17
KR20230110368A (ko) 2023-07-21
DK3302709T3 (da) 2021-08-23
EP3302709A4 (en) 2019-01-23
WO2016196539A3 (en) 2017-02-16
PL3302709T3 (pl) 2021-12-06
US20180296649A1 (en) 2018-10-18
EA201890012A1 (ru) 2018-05-31
EP3302709B1 (en) 2021-05-19
CN108025188A (zh) 2018-05-11
WO2016196539A2 (en) 2016-12-08
US11298410B2 (en) 2022-04-12
JP6968416B2 (ja) 2021-11-17
EP3302709A2 (en) 2018-04-11
JP2018516572A (ja) 2018-06-28
EP3919622A1 (en) 2021-12-08
AU2016270702B2 (en) 2022-01-20
AU2016270702A1 (en) 2017-12-21

Similar Documents

Publication Publication Date Title
KR102553518B1 (ko) Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
AU2017219605B2 (en) Excision of retroviral nucleic acid sequences
JP7118588B2 (ja) Ｈｉｖ感染のｒｎａガイド処置のための方法および組成物
US20190367910A1 (en) Methods and compositions for rna-guided treatment of hiv infection
JP2019517503A (ja) 遺伝子編集戦略によるｈｉｖ−１のネガティブフィードバック調節
HK1253882B (en) Methods and compositions for rna-guided treatment of hiv infection
CN108148875B (zh) 一种基因编辑系统、表达载体、基因编辑试剂盒及其用途
HK40000290A (en) Excision of retroviral nucleic acid sequences

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20171228

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20200623

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20220610

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20230405

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20230705

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20230705

End annual number: 3

Start annual number: 1

PG1601 Publication of registration