JP2006518996A - 心筋収縮性をｉｎｖｉｖｏで調節するための方法 - Google Patents

心筋収縮性をｉｎｖｉｖｏで調節するための方法 Download PDF

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JP2006518996A
JP2006518996A JP2006500911A JP2006500911A JP2006518996A JP 2006518996 A JP2006518996 A JP 2006518996A JP 2006500911 A JP2006500911 A JP 2006500911A JP 2006500911 A JP2006500911 A JP 2006500911A JP 2006518996 A JP2006518996 A JP 2006518996A
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serca2
expression
gene
cells
dna
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ディルマン、ウルフガング、エイチ．
ジョルダーノ、フランク
メストリル、ルーベン
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University of California San Diego UCSD
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03008—Ca2+-transporting ATPase (3.6.3.8)
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Electrotherapy Devices (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2006500911A 2003-01-13 2004-01-12 心筋収縮性をｉｎｖｉｖｏで調節するための方法 Pending JP2006518996A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US10/342,120 US7745416B2 (en) 1995-04-11 2003-01-13 Method for in vivo regulation of cardiac muscle contractility
PCT/US2004/000719 WO2004062618A2 (en) 2003-01-13 2004-01-12 Method for in vivo regulation of cardiac muscle contractility

Publications (2)

Publication Number Publication Date
JP2006518996A true JP2006518996A (ja) 2006-08-24
JP2006518996A5 JP2006518996A5 (https=) 2007-03-01

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JP2006500911A Pending JP2006518996A (ja) 2003-01-13 2004-01-12 心筋収縮性をｉｎｖｉｖｏで調節するための方法

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Country Link
US (1) US7745416B2 (https=)
EP (1) EP1590001B1 (https=)
JP (1) JP2006518996A (https=)
AT (1) ATE416792T1 (https=)
AU (2) AU2004204815B2 (https=)
CA (1) CA2513249A1 (https=)
DE (1) DE602004018269D1 (https=)
ES (1) ES2318264T3 (https=)
IL (1) IL169663A (https=)
WO (1) WO2004062618A2 (https=)

Cited By (20)

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JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのＣＲＩＳＰＲ−Ｃａｓ系および組成物の送達、使用および治療上の適用
JP2016523082A (ja) * 2013-06-17 2016-08-08 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
JP2016524472A (ja) * 2013-06-17 2016-08-18 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
JP2017527256A (ja) * 2013-12-12 2017-09-21 ザ・ブロード・インスティテュート・インコーポレイテッド ＨＢＶ及びウイルス性疾患及び障害のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
US10494621B2 (en) 2015-06-18 2019-12-03 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US10550372B2 (en) 2013-12-12 2020-02-04 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US10696986B2 (en) 2014-12-12 2020-06-30 The Board Institute, Inc. Protected guide RNAS (PGRNAS)
US10711285B2 (en) 2013-06-17 2020-07-14 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US10781444B2 (en) 2013-06-17 2020-09-22 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10851357B2 (en) 2013-12-12 2020-12-01 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US10930367B2 (en) 2012-12-12 2021-02-23 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
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US11155795B2 (en) 2013-12-12 2021-10-26 The Broad Institute, Inc. CRISPR-Cas systems, crystal structure and uses thereof
US11407985B2 (en) 2013-12-12 2022-08-09 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing
US11578312B2 (en) 2015-06-18 2023-02-14 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
JP2023513211A (ja) * 2020-02-07 2023-03-30 ユニバーシティ オブ ロチェスター タンパク質合成を増強するためのＣＲＩＳＰＲ－Ｃａｓ１３による標的ＲＮＡ翻訳
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12435320B2 (en) 2014-12-24 2025-10-07 The Broad Institute, Inc. CRISPR having or associated with destabilization domains
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof

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US20050095227A1 (en) * 1997-07-22 2005-05-05 The General Hospital Corporation Treating heart failure
WO2005023292A1 (en) 2003-09-03 2005-03-17 The General Hospital Corporation Methods of treating restenosis
CN101056539B (zh) * 2004-09-09 2012-12-12 综合医院公司 调节心脏细胞中的磷酸酶活性
WO2006089340A2 (en) * 2005-02-23 2006-08-31 V-Kardia Pty Ltd Polynucleotide delivery to cardiac tissue
KR20090035711A (ko) * 2006-07-25 2009-04-10 셀라돈 코포레이션 유전자 치료를 위한 아데노-관련 바이러스 벡터의 전방 심외막 관상에의 연장 주입
EP2164330A4 (en) * 2007-06-18 2011-12-28 Celladon Corp SERCA2-BASED THERAPEUTIC COMPOSITIONS AND METHODS OF USE
DK2205618T3 (en) * 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
KR20100129295A (ko) 2008-02-19 2010-12-08 셀라돈 코포레이션 심근 내 바이러스 벡터의 흡수를 개선시키기 위한 조성물
US20180099029A9 (en) * 2010-04-15 2018-04-12 Mount Sinai School Of Medicine Serca2 therapeutic compositions and methods of use

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WO2000038518A1 (en) * 1998-12-28 2000-07-06 Arch Development Corporation Efficient and stable (in vivo) gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors

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WO1996032139A1 (en) * 1995-04-11 1996-10-17 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
WO2000015821A1 (en) * 1998-09-11 2000-03-23 The Regents Of The University Of California Recombinant adenovirus for tissue specific expression in heart
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Cited By (38)

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US12252707B2 (en) 2012-12-12 2025-03-18 The Broad Institute, Inc. Delivery, Engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US10930367B2 (en) 2012-12-12 2021-02-23 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
US12441995B2 (en) 2013-06-17 2025-10-14 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10781444B2 (en) 2013-06-17 2020-09-22 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10577630B2 (en) 2013-06-17 2020-03-03 The Broad Institute, Inc. Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
JP2020063238A (ja) * 2013-06-17 2020-04-23 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
JP2016523082A (ja) * 2013-06-17 2016-08-08 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
US10711285B2 (en) 2013-06-17 2020-07-14 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのＣＲＩＳＰＲ−Ｃａｓ系および組成物の送達、使用および治療上の適用
US12018275B2 (en) 2013-06-17 2024-06-25 The Broad Institute, Inc. Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US12553065B2 (en) 2013-06-17 2026-02-17 The Broad Institute, Inc. Optimized CRISPR-CAS double nickase systems, methods and compositions for sequence manipulation
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
JP2016524472A (ja) * 2013-06-17 2016-08-18 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
US11407985B2 (en) 2013-12-12 2022-08-09 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12258595B2 (en) 2013-12-12 2025-03-25 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US11591581B2 (en) 2013-12-12 2023-02-28 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11597919B2 (en) 2013-12-12 2023-03-07 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
JP2017527256A (ja) * 2013-12-12 2017-09-21 ザ・ブロード・インスティテュート・インコーポレイテッド ＨＢＶ及びウイルス性疾患及び障害のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
US10550372B2 (en) 2013-12-12 2020-02-04 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US12251450B2 (en) 2013-12-12 2025-03-18 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
US10851357B2 (en) 2013-12-12 2020-12-01 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11155795B2 (en) 2013-12-12 2021-10-26 The Broad Institute, Inc. CRISPR-Cas systems, crystal structure and uses thereof
US12410435B2 (en) 2013-12-12 2025-09-09 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US10696986B2 (en) 2014-12-12 2020-06-30 The Board Institute, Inc. Protected guide RNAS (PGRNAS)
US11624078B2 (en) 2014-12-12 2023-04-11 The Broad Institute, Inc. Protected guide RNAS (pgRNAS)
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AU2004204815A1 (en) 2004-07-29
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