CA2849920A1 - Methods and compositions for regulating hiv infection - Google Patents

Methods and compositions for regulating hiv infection Download PDF

Info

Publication number
CA2849920A1
CA2849920A1 CA 2849920 CA2849920A CA2849920A1 CA 2849920 A1 CA2849920 A1 CA 2849920A1 CA 2849920 CA2849920 CA 2849920 CA 2849920 A CA2849920 A CA 2849920A CA 2849920 A1 CA2849920 A1 CA 2849920A1
Authority
CA
Canada
Prior art keywords
cell
hiv
gene
sequence
dna
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.)
Abandoned
Application number
CA 2849920
Other languages
English (en)
French (fr)
Inventor
Michael C. Holmes
Philip D. Gregory
Fyodor Urnov
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Biosciences 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 Sangamo Biosciences Inc filed Critical Sangamo Biosciences Inc
Publication of CA2849920A1 publication Critical patent/CA2849920A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2/00Peptides of undefined number of amino acids; Derivatives thereof
    • 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
    • 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
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004Type II site-specific deoxyribonuclease (3.1.21.4)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • AIDS & HIV (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Communicable Diseases (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA 2849920 2011-10-06 2012-10-04 Methods and compositions for regulating hiv infection Abandoned CA2849920A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161544101P 2011-10-06 2011-10-06
US61/544,101 2011-10-06
PCT/US2012/058775 WO2013052681A1 (en) 2011-10-06 2012-10-04 Methods and compositions for regulating hiv infection

Publications (1)

Publication Number Publication Date
CA2849920A1 true CA2849920A1 (en) 2013-04-11

Family

ID=48044158

Family Applications (1)

Application Number Title Priority Date Filing Date
CA 2849920 Abandoned CA2849920A1 (en) 2011-10-06 2012-10-04 Methods and compositions for regulating hiv infection

Country Status (7)

Country Link
US (2) US20130171732A1 (OSRAM)
EP (1) EP2764102A4 (OSRAM)
JP (1) JP2014530603A (OSRAM)
AU (1) AU2012318562A1 (OSRAM)
CA (1) CA2849920A1 (OSRAM)
HK (1) HK1200491A1 (OSRAM)
WO (1) WO2013052681A1 (OSRAM)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2852955C (en) * 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
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
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、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
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
EP3011030B1 (en) * 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
AU2014350051A1 (en) * 2013-11-18 2016-07-07 Crispr Therapeutics Ag CRISPR-Cas system materials and methods
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Hbv 및 바이러스 질병 및 질환을 위한 crispr­cas 시스템 및 조성물의 전달,용도 및 치료적 적용
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2016014837A1 (en) * 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的crispr酶突變

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1352960A1 (en) * 2002-04-12 2003-10-15 Viruvation B.V. Antiviral therapy on the basis of RNA interference
US20100055793A1 (en) * 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) * 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US20080003681A1 (en) * 2006-06-28 2008-01-03 Mahalaxmi Gita Bangera Methods for altering cellular susceptibility to infection
US8563314B2 (en) * 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2303292A4 (en) * 2008-06-09 2013-01-02 New York Medical College COMPOSITIONS WITH CARDIAL STEM CELLS WITH OVEREXPRESSION OF SPECIFIC MICRORNAS AND METHOD FOR THEIR USE IN THE REPAIR OF THE CARED MYOCARDIC
WO2010107493A2 (en) * 2009-03-20 2010-09-23 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
ES2550202T3 (es) * 2009-08-03 2015-11-05 Recombinetics, Inc. Métodos y composiciones para la modificación dirigida de genes
CN102071219B (zh) * 2010-11-05 2014-03-12 冯小荣 改腺载体及其在艾滋病预防和治疗药物中的应用

Also Published As

Publication number Publication date
JP2014530603A (ja) 2014-11-20
WO2013052681A1 (en) 2013-04-11
EP2764102A1 (en) 2014-08-13
US20130171732A1 (en) 2013-07-04
AU2012318562A1 (en) 2014-04-10
HK1200491A1 (en) 2015-08-07
EP2764102A4 (en) 2015-06-10
US20150267223A1 (en) 2015-09-24

Similar Documents

Publication Publication Date Title
US20230203540A1 (en) Methods and compositions for nuclease-mediated targeted integration of transgenes into mammalian liver cells
JP6629917B2 (ja) 遺伝子疾患の治療のための方法および組成物
CA2849920A1 (en) Methods and compositions for regulating hiv infection
US9833479B2 (en) Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US9566352B2 (en) Methods and compositions for inhibiting viral entry into cells
US9757420B2 (en) Gene editing for HIV gene therapy
CA2931637C (en) Methods and compositions for treating hemophilia
CA2852955C (en) Methods and compositions for modification of the hprt locus
DK3196301T3 (en) METHODS AND COMPOSITIONS FOR TREATING MONOGENIC DISEASES
JP2021010383A (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
JP2022105621A (ja) 造血幹細胞および前駆細胞におけるscid関連遺伝子の遺伝子修正
HK40012091B (en) Compositions for inhibiting viral entry into cells
HK40012091A (en) Compositions for inhibiting viral entry into cells
HK1187374B (en) Compositions for inhibiting viral entry into cells
HK1187374A (en) Compositions for inhibiting viral entry into cells

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20171004