PL3302709T3 - Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego - Google Patents

Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego

Info

Publication number
PL3302709T3
PL3302709T3 PL16804274T PL16804274T PL3302709T3 PL 3302709 T3 PL3302709 T3 PL 3302709T3 PL 16804274 T PL16804274 T PL 16804274T PL 16804274 T PL16804274 T PL 16804274T PL 3302709 T3 PL3302709 T3 PL 3302709T3
Authority
PL
Poland
Prior art keywords
homing
rna
compositions
methods
hiv infection
Prior art date
Application number
PL16804274T
Other languages
English (en)
Inventor
Kamel Khalili
Wenhui Hu
Yonggang Zhang
Original Assignee
Temple University - Of The Commonwealth System Of Higher Education
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 Temple University - Of The Commonwealth System Of Higher Education filed Critical Temple University - Of The Commonwealth System Of Higher Education
Publication of PL3302709T3 publication Critical patent/PL3302709T3/pl

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] 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
    • 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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • AIDS & HIV (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL16804274T 2015-06-01 2016-06-01 Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego PL3302709T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562169384P 2015-06-01 2015-06-01
US201562169633P 2015-06-02 2015-06-02
US201662308320P 2016-03-15 2016-03-15
EP16804274.5A EP3302709B1 (en) 2015-06-01 2016-06-01 Methods and compositions for rna-guided treatment of hiv infection
PCT/US2016/035141 WO2016196539A2 (en) 2015-06-01 2016-06-01 Methods and compositions for rna-guided treatment of hiv infection

Publications (1)

Publication Number Publication Date
PL3302709T3 true PL3302709T3 (pl) 2021-12-06

Family

ID=57442165

Family Applications (1)

Application Number Title Priority Date Filing Date
PL16804274T PL3302709T3 (pl) 2015-06-01 2016-06-01 Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego

Country Status (15)

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

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
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
US9925248B2 (en) 2013-08-29 2018-03-27 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EA037359B1 (ru) 2015-06-01 2021-03-17 Тэмпл Юниверсити - Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич
EP3356521A4 (en) * 2015-09-28 2019-03-13 Temple University - Of The Commonwealth System of Higher Education METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したＣａｓ９蛋白質
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 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
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US20200140865A1 (en) * 2017-04-17 2020-05-07 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
WO2019010422A1 (en) * 2017-07-07 2019-01-10 The Broad Institute, Inc. ANTIVIRAL THERAPY BASED ON THE CRISPR SYSTEM
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 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
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
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
CN115279898A (zh) * 2019-10-23 2022-11-01 成对植物服务股份有限公司 用于植物中rna模板化编辑的组合物和方法
EP4114952A4 (en) * 2020-03-05 2024-05-08 Board of Regents of the University of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
EP4179120A4 (en) * 2020-07-13 2024-08-21 The United States Government as represented by the Department of Veterans Affairs Methods and compositions for crispr/cas9 guide rna efficiency and specificity against genetically diverse hiv-1 isolates
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
WO2022198229A1 (en) * 2021-03-17 2022-09-22 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

Family Cites Families (58)

* 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
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
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
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
WO2014093718A1 (en) 2012-12-12 2014-06-19 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
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2895155C (en) 2012-12-17 2021-07-06 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
JP6576904B2 (ja) * 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ ＨＩＶ−１プロウイルスＤＮＡのｉｎｖｉｖｏ切除のための組成物及び方法
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP4596565A3 (en) 2013-06-04 2025-11-05 President And Fellows Of Harvard College Rna-guided transcriptional regulation
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
JP7120717B2 (ja) 2013-07-09 2022-08-17 プレジデント アンド フェローズ オブ ハーバード カレッジ 多重ｒｎａ誘導型ゲノム編集
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US10563225B2 (en) 2013-07-26 2020-02-18 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
US9925248B2 (en) 2013-08-29 2018-03-27 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
WO2016022363A2 (en) 2014-07-30 2016-02-11 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
EA037359B1 (ru) 2015-06-01 2021-03-17 Тэмпл Юниверсити - Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич
EP4159856A1 (en) 2015-06-18 2023-04-05 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3356521A4 (en) 2015-09-28 2019-03-13 Temple University - Of The Commonwealth System of Higher Education METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
US20190083656A1 (en) 2015-10-16 2019-03-21 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing cpf1 for rna-guided gene editing
WO2017070284A1 (en) 2015-10-21 2017-04-27 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating hepatitis b virus
EP3387001A4 (en) 2015-12-09 2019-08-14 Excision Biotherapeutics, Inc. GENETIZATION METHOD AND AGENT TO ELIMINATE THE RISK OF JC VIRUS ACTIVATION AND PML (PROGRESSIVE MULTIFOCALER LEUKOENCEEPHALOPATHY) DURING IMMUNOSUPPRESSIVE THERAPY
AU2017211062A1 (en) 2016-01-25 2018-06-07 Excision Biotherapeutics Methods and compositions for RNA-guided treatment of HIV infection
MX2018009904A (es) 2016-02-15 2019-07-08 Univ Temple Escision de secuencias de acido nucleico retrovirales.
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
WO2017213898A2 (en) 2016-06-07 2017-12-14 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
CA3038960A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
WO2018071623A2 (en) 2016-10-12 2018-04-19 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
WO2018140269A1 (en) 2017-01-26 2018-08-02 Excision Biotherapeutics, Inc. Lentivirus and non-integrating lentivirus as viral vector to deliver crispr therapeutic
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
US20200140865A1 (en) 2017-04-17 2020-05-07 Temple University - Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents

Also Published As

Publication number Publication date
HK1253882A1 (en) 2019-07-05
AU2016270702B2 (en) 2022-01-20
KR20230110368A (ko) 2023-07-21
SG10201914137YA (en) 2020-02-27
US12251429B2 (en) 2025-03-18
WO2016196539A2 (en) 2016-12-08
WO2016196539A3 (en) 2017-02-16
MX2017015582A (es) 2018-09-06
JP6968416B2 (ja) 2021-11-17
ES2886480T3 (es) 2021-12-20
AU2016270702A1 (en) 2017-12-21
CA2987927C (en) 2024-03-19
ZA201708236B (en) 2019-12-18
US11298410B2 (en) 2022-04-12
CN108025188A (zh) 2018-05-11
EA201890012A1 (ru) 2018-05-31
EP3302709A4 (en) 2019-01-23
KR102553518B1 (ko) 2023-07-07
AU2025204187A1 (en) 2025-06-26
CA2987927A1 (en) 2016-12-08
KR20180023911A (ko) 2018-03-07
EP3919622A1 (en) 2021-12-08
US11273209B2 (en) 2022-03-15
EP3302709B1 (en) 2021-05-19
US20200282025A1 (en) 2020-09-10
US20210052709A1 (en) 2021-02-25
EA037359B1 (ru) 2021-03-17
US20180296649A1 (en) 2018-10-18
EP3302709A2 (en) 2018-04-11
DK3302709T3 (da) 2021-08-23
JP2018516572A (ja) 2018-06-28
AU2022201875A1 (en) 2022-04-07

Similar Documents

Publication Publication Date Title
PL3302709T3 (pl) Sposoby i kompozycje do leczenia zakażenia HIV przy użyciu RNA naprowadzającego
IL282881A (en) Methods for treating hepatitis b infection
IL280459A (en) Phosphoramidates for the treatment of hepatitis b virus
IL261082A (en) Compositions and methods for modifying genomes
LT3356529T (lt) Kompozicijos ir būdai, skirti lpa genų raiškos slopinimui
IL257384A (en) Preparations containing rnai and their use for the treatment of infection with the hepatitis b virus
IL259077A (en) Vaccines against hepatitis b virus
DK3400291T3 (da) Manipuleret virus
EP3139954A4 (en) Methods and compositions for treating hepatitis b virus infections
DK3433368T3 (da) Transreplikerende rna
DK3390631T3 (da) Fremgangsmåder og sammensætninger til t-rna-baseret guide-rna-ekspression
DK3390632T3 (da) Fremgangsmåder og sammensætninger til polymerase ii (pol-ii)-baseret guide-rna-ekspression
EP3313989C0 (en) MODIFIED CRISPR RNA AND MODIFIED SINGLE CRISPR RNA AND USES THEREOF
IL251450A0 (en) Preparations and methods for silencing hepatitis b virus gene expression
MX373703B (es) Derivados de azepano y metodos para tratar infecciones por hepatitis b.
MX383929B (es) Derivados y métodos de tratamiento de las infecciones por hepatitis b.
DK3872085T5 (da) Stam-virussekvenser og anvendelser heraf
KR20180084891A (ko) 구조 조성물 및 방법
DK3234134T3 (da) Målrettet rna-redigering
DK3366295T3 (da) Fremgangsmåder til behandling af filoviridae virusinfektioner
DK3265102T3 (da) Beta-d-2'-deoxy-2'alpha-fluoro-2'-beta-c-substituerede-2-modificerede-n6-substituerede purinnukleotider til hcv-behandling
DK3512863T3 (da) 2¿-substituerede-N6-substituerede purinnukleotider til behandling af RNA-virus
FI20155008A7 (fi) Ajoneuvoavusteinen työlaite
DK3137078T3 (da) Behandling af hepatitis delta-virusinfektion
EP3346006A4 (en) METHOD FOR DNA AMPLIFICATION