WO2012158985A3 - Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms - Google Patents

Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms Download PDF

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Publication number
WO2012158985A3
WO2012158985A3 PCT/US2012/038464 US2012038464W WO2012158985A3 WO 2012158985 A3 WO2012158985 A3 WO 2012158985A3 US 2012038464 W US2012038464 W US 2012038464W WO 2012158985 A3 WO2012158985 A3 WO 2012158985A3
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WO
WIPO (PCT)
Prior art keywords
cells
zfn
site
methods
zinc finger
Prior art date
Application number
PCT/US2012/038464
Other languages
French (fr)
Other versions
WO2012158985A2 (en
Inventor
Eric M. Ostertag
John S. CRAWFORD
J. Keith Joung
Franklin Kent HAMRA
Original Assignee
Transposagen Biopharmaceuticals, Inc.
The General Hospital Corporation
The Board Of Regents Of The University Of Texas System
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.)
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Publication date
Application filed by Transposagen Biopharmaceuticals, Inc., The General Hospital Corporation, The Board Of Regents Of The University Of Texas System filed Critical Transposagen Biopharmaceuticals, Inc.
Publication of WO2012158985A2 publication Critical patent/WO2012158985A2/en
Publication of WO2012158985A3 publication Critical patent/WO2012158985A3/en

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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/108—Swine

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Environmental Sciences (AREA)
  • Mycology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Methods for site-specific genetic engineering using zinc finger nuclease (ZFN) of stem cells and gametes, including but not limited to pluripotent cells, totipotent cells, somatic stem cells, spermatogonial stem cells (SSCs), embryonic stem (ES) cells, induced pluripotent stem (iPS) cells, embryos, germ cells, primordial germ cells (PGCs), plant tube cells, pollen cells, and spores are presented herein.
PCT/US2012/038464 2011-05-17 2012-05-17 Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms WO2012158985A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161487270P 2011-05-17 2011-05-17
US61/487,270 2011-05-17

Publications (2)

Publication Number Publication Date
WO2012158985A2 WO2012158985A2 (en) 2012-11-22
WO2012158985A3 true WO2012158985A3 (en) 2013-03-21

Family

ID=47177646

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/038464 WO2012158985A2 (en) 2011-05-17 2012-05-17 Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms

Country Status (1)

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WO (1) WO2012158985A2 (en)

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EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20160046959A1 (en) * 2013-03-15 2016-02-18 Carlisle P. Landel Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
US20140359796A1 (en) * 2013-05-31 2014-12-04 Recombinetics, Inc. Genetically sterile animals
GB201310031D0 (en) 2013-06-05 2013-07-17 Pirbright Inst The Cell
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9834791B2 (en) 2013-11-07 2017-12-05 Editas Medicine, Inc. CRISPR-related methods and compositions with governing gRNAS
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US20150275231A1 (en) * 2014-03-31 2015-10-01 Mice With Horns, Llc Method of Preventing or Reducing Virus Transmission in Animals
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CN108513575A (en) 2015-10-23 2018-09-07 哈佛大学的校长及成员们 Nucleobase editing machine and application thereof
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (en) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 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
JP2020510439A (en) 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 Base-editing factor from cytosine to guanine
SG11201908658TA (en) 2017-03-23 2019-10-30 Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN111757937A (en) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 Use of adenosine base editor
CN108378019B (en) * 2018-03-13 2021-03-02 诺赛联合(北京)生物医学科技有限公司 Cryopreservation liquid for human spermatogonial stem cells
DE112020001342T5 (en) 2019-03-19 2022-01-13 President and Fellows of Harvard College Methods and compositions for editing nucleotide sequences
EP4146804A1 (en) 2020-05-08 2023-03-15 The Broad Institute Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence

Citations (4)

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US20070180542A1 (en) * 2004-04-12 2007-08-02 The Trustees Of The University Of Pennsylvania Culture conditions and growth factors affecting fate determination, self-renewal and expansion of rat spermatogonial stem cells
WO2010065550A2 (en) * 2008-12-01 2010-06-10 Board Of Regents University Of Texas System Production and use of rat spermatogonial stem cell lines
US20100287628A1 (en) * 2009-04-23 2010-11-11 Ostertag Eric M Genetically Modified Rat Models for Cancer
US20110061118A1 (en) * 2008-03-17 2011-03-10 Helmholtz Zentrum Munchen Vectors and methods for generating vector-free induced pluripotent stem (ips) cells using site-specific recombination

Patent Citations (4)

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US20070180542A1 (en) * 2004-04-12 2007-08-02 The Trustees Of The University Of Pennsylvania Culture conditions and growth factors affecting fate determination, self-renewal and expansion of rat spermatogonial stem cells
US20110061118A1 (en) * 2008-03-17 2011-03-10 Helmholtz Zentrum Munchen Vectors and methods for generating vector-free induced pluripotent stem (ips) cells using site-specific recombination
WO2010065550A2 (en) * 2008-12-01 2010-06-10 Board Of Regents University Of Texas System Production and use of rat spermatogonial stem cell lines
US20100287628A1 (en) * 2009-04-23 2010-11-11 Ostertag Eric M Genetically Modified Rat Models for Cancer

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