JP2002535994A - 標的ｄｎａのインビボ除去による遺伝子修復 - Google Patents

標的ｄｎａのインビボ除去による遺伝子修復

Info

Publication number
JP2002535994A
JP2002535994A JP2000597444A JP2000597444A JP2002535994A JP 2002535994 A JP2002535994 A JP 2002535994A JP 2000597444 A JP2000597444 A JP 2000597444A JP 2000597444 A JP2000597444 A JP 2000597444A JP 2002535994 A JP2002535994 A JP 2002535994A
Authority
JP
Japan
Prior art keywords
dna
vector
restriction endonuclease
target dna
cell
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.)
Pending
Application number
JP2000597444A
Other languages
English (en)
Japanese (ja)
Other versions
JP2002535994A5 (https=
Inventor
シューリカ，アンドレ
マリガン，リチャード，シー．
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.)
Institut Pasteur
Original Assignee
Institut Pasteur
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 Institut Pasteur filed Critical Institut Pasteur
Publication of JP2002535994A publication Critical patent/JP2002535994A/ja
Publication of JP2002535994A5 publication Critical patent/JP2002535994A5/ja
Pending legal-status Critical Current

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
    • C12N15/87—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2000597444A 1999-02-03 2000-02-03 標的ｄｎａのインビボ除去による遺伝子修復 Pending JP2002535994A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11847299P 1999-02-03 1999-02-03
US60/118,472 1999-02-03
PCT/US2000/002949 WO2000046385A1 (en) 1999-02-03 2000-02-03 Gene repair involving in vivo excision of targeting dna

Publications (2)

Publication Number Publication Date
JP2002535994A true JP2002535994A (ja) 2002-10-29
JP2002535994A5 JP2002535994A5 (https=) 2007-04-05

Family

ID=22378818

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2000597444A Pending JP2002535994A (ja) 1999-02-03 2000-02-03 標的ｄｎａのインビボ除去による遺伝子修復

Country Status (6)

Country Link
US (4) US20020110898A1 (https=)
EP (1) EP1151124A1 (https=)
JP (1) JP2002535994A (https=)
AU (1) AU2982300A (https=)
CA (1) CA2360878A1 (https=)
WO (1) WO2000046385A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006518372A (ja) * 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7102055B1 (en) * 1997-11-18 2006-09-05 Pioneer Hi-Bred International, Inc. Compositions and methods for the targeted insertion of a nucleotide sequence of interest into the genome of a plant
US6187994B1 (en) * 1997-11-18 2001-02-13 Pioneer Hi-Bred International, Inc. Compositions and methods for genetic modification of plants
JP2002535994A (ja) * 1999-02-03 2002-10-29 ザ チルドレンズ メディカル センター コーポレイション 標的ｄｎａのインビボ除去による遺伝子修復
WO2000046386A2 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
AU2002339224B2 (en) 2001-09-14 2008-10-09 Kuros Us Llc Packaging of immunostimulatory substances into virus-like particles: method of preparation and use
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20060078552A1 (en) 2002-03-15 2006-04-13 Sylvain Arnould Hybrid and single chain meganucleases and use thereof
WO2009095742A1 (en) 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
US20030232410A1 (en) * 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
US11311574B2 (en) 2003-08-08 2022-04-26 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
BRPI0612862A2 (pt) * 2005-07-18 2010-11-30 Pioneer Hi Bred Int polinucleotìdeo isolado, método para obtenção de uma célula transformada, método para obtenção de uma planta transformada, método de produção de recombinação de dna de sìtio especìfico, biblioteca isolada, método de geração de biblioteca de moléculas e método para selecionar sìtio de recombinação frt modificado
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
US7852247B2 (en) * 2006-12-05 2010-12-14 Texas Instruments Incorporated Mixed-signal filter
CN101849010A (zh) * 2007-06-29 2010-09-29 先锋高级育种国际公司 用于改变单子叶植物细胞的基因组的方法
EP2215252A4 (en) * 2007-12-07 2011-01-26 Prec Biosciences Inc RATIONAL-DESIGNED MEGANUCLEASES WITH DETECTION SEQUENCES FROM DNASE-HYPERSENSITIVE REGIONS OF THE HUMAN GENOME
WO2009114321A2 (en) * 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
US20100071083A1 (en) * 2008-03-12 2010-03-18 Smith James J Temperature-dependent meganuclease activity
EP2279250A4 (en) * 2008-04-28 2011-10-12 Prec Biosciences Inc Fusion Molecules of DNA Binding Proteins with Rational Design and Effects Domain
DK3211075T3 (en) 2008-07-14 2019-01-21 Prec Biosciences Inc RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10508478A (ja) * 1994-11-07 1998-08-25 アンスティテュ・パストゥール Ｉ−Ｓｃｅ Ｉ酵素をコードするヌクレオチド配列、及びその使用
US5830729A (en) * 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US285538A (en) * 1883-09-25 Ore-crusher
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5474896A (en) 1992-05-05 1995-12-12 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
ES2132330T3 (es) 1993-12-20 1999-08-16 Akzo Nobel Nv Vacuna para la proteccion de caballos frente a la infeccion por el virus del herpes equino.
WO2000046386A2 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
JP2002535994A (ja) 1999-02-03 2002-10-29 ザ チルドレンズ メディカル センター コーポレイション 標的ｄｎａのインビボ除去による遺伝子修復
ATE466088T1 (de) * 2001-09-14 2010-05-15 Cellectis Zufällige integration von polynukleotide nach in vivo linearisierung
JP3915752B2 (ja) * 2003-07-25 2007-05-16 船井電機株式会社 デジタル情報記録装置およびそれを備える映像記録再生装置

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10508478A (ja) * 1994-11-07 1998-08-25 アンスティテュ・パストゥール Ｉ−Ｓｃｅ Ｉ酵素をコードするヌクレオチド配列、及びその使用
US5830729A (en) * 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JPN5002003148, ＣＯＨＥＮ−ＴＡＮＮＯＵＤＪＩ Ｍ, ＭＯＬＥＣＵＬＡＲ ＡＮＤ ＣＥＬＬＵＬＡＲ ＢＩＯＬＯＧＹ, 199803, Ｖ１８ Ｎ３, Ｐ１４４４−１４４８ *
JPN5002003149, ＣＨＯＵＬＩＫＡ Ａ, ＭＯＬＥＣＵＬＡＲ ＡＮＤ ＣＥＬＬＵＬＡＲ ＢＩＯＬＯＧＹ, 199504, Ｖ１５ Ｎ４, Ｐ１９６８−１９７３, US *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006518372A (ja) * 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用

Also Published As

Publication number Publication date
US7285538B2 (en) 2007-10-23
US20090060896A1 (en) 2009-03-05
WO2000046385A1 (en) 2000-08-10
EP1151124A1 (en) 2001-11-07
US7960525B2 (en) 2011-06-14
US20030229039A1 (en) 2003-12-11
US20020110898A1 (en) 2002-08-15
AU2982300A (en) 2000-08-25
US20120165400A1 (en) 2012-06-28
CA2360878A1 (en) 2000-08-10

Similar Documents

Publication Publication Date Title
US7960525B2 (en) Gene repair involving in vivo excision of targeting DNA
US9458439B2 (en) Chromosomal modification involving the induction of double-stranded DNA cleavage and homologous recombination at the cleavage site
TW202028461A (zh) 核酸構築體及使用方法
JP2019525756A (ja) Ｃｐｆ１に基づくゲノム編集の治療適用
JP2022529424A (ja) ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにしたゲノム編集組成物
JP2023522788A (ja) 標的化されたゲノム組込みによってデュシェンヌ型筋ジストロフィーを矯正するためのｃｒｉｓｐｒ／ｃａｓ９療法
JP2023525007A (ja) 転位に基づく療法
KR20020057953A (ko) 진핵 세포에서의 서열 특이적 ｄｎａ 재조합
JP7667595B2 (ja) Ａｑｐ１ ＲＮＡを標的とするｓｇＲＮＡ及びそのベクターと使用
US20250002876A1 (en) Mobile elements and chimeric constructs thereof
JP7161730B2 (ja) 顆粒状角膜変性症に対する遺伝子治療薬
EP3640334A1 (en) Genome editing system for repeat expansion mutation
JP2025106359A (ja) 細胞の遺伝子改変のための発現構築物
CN114144231A (zh) 用于治疗癌症的crispr方法
AU2004202860B2 (en) Gene repair involving in vivo excision of targeting DNA
US20250269057A1 (en) Therapeutic lama2 payload for treatment of congenital muscular dystrophy

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20070123

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20070123

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20091125

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20100222

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20100301

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20100323

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20100330

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20100423

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20100506

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100525

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20100902