JP2005528912A - 幹細胞におけるスプライセオソーム介在rnaトランススプライシング - Google Patents

幹細胞におけるスプライセオソーム介在rnaトランススプライシング Download PDF

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JP2005528912A
JP2005528912A JP2004511476A JP2004511476A JP2005528912A JP 2005528912 A JP2005528912 A JP 2005528912A JP 2004511476 A JP2004511476 A JP 2004511476A JP 2004511476 A JP2004511476 A JP 2004511476A JP 2005528912 A JP2005528912 A JP 2005528912A
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nucleic acid
acid molecule
stem cell
mrna
target
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JP2005528912A5 (enExample
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ミッチェル,ロイド,ジー.
アングルハード,ジョン
リュウ,シャオ,ミン
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イントロン,インコーポレーテッド
ユニバーシティー オブ アイオワ リサーチ ファンデーション
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Publication of JP2005528912A publication Critical patent/JP2005528912A/ja
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    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0688Cells from the lungs or the respiratory tract
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N2510/00Genetically modified cells
    • C12N2510/02Cells for production

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Pulmonology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2004511476A 2002-06-05 2003-06-05 幹細胞におけるスプライセオソーム介在rnaトランススプライシング Pending JP2005528912A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US38659402P 2002-06-05 2002-06-05
PCT/US2003/017954 WO2003104416A2 (en) 2002-06-05 2003-06-05 Spliceosome mediated rna trans-splicing in stem cells

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JP2005528912A true JP2005528912A (ja) 2005-09-29
JP2005528912A5 JP2005528912A5 (enExample) 2006-03-02

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JP2004511476A Pending JP2005528912A (ja) 2002-06-05 2003-06-05 幹細胞におけるスプライセオソーム介在rnaトランススプライシング

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US (1) US20040180429A1 (enExample)
EP (1) EP1549755A4 (enExample)
JP (1) JP2005528912A (enExample)
AU (1) AU2003247505A1 (enExample)
CA (1) CA2488249A1 (enExample)
WO (1) WO2003104416A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116171326A (zh) * 2020-07-17 2023-05-26 绿光生物科技股份有限公司 用于基因病症的核酸疗法

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2005207053A1 (en) * 2004-01-23 2005-08-04 Virxsys Corporation Expression of ApoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
EP2151248A1 (en) * 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
US10987433B2 (en) 2015-11-19 2021-04-27 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
EP3781213A4 (en) 2018-04-17 2022-10-19 The Trustees of the University of Pennsylvania TRANS-SPLICE MOLECULES
CN118786214A (zh) * 2022-01-18 2024-10-15 新加坡国立大学 反式剪接RNA(tsRNA)
CA3252043A1 (en) 2022-05-13 2023-11-16 Ascidian Therapeutics, Inc. ABCA4 TRANS-SPLICING MOLECULES

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ241311A (en) * 1991-01-17 1995-03-28 Gen Hospital Corp Rna sequence having trans-splicing activity, plant strains
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US6083702A (en) * 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
AU730305B2 (en) * 1995-12-15 2001-03-01 Intronn Llc Therapeutic molecules generated by trans-splicing
CA2278734A1 (en) * 1997-01-23 1998-07-30 Immusol Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116171326A (zh) * 2020-07-17 2023-05-26 绿光生物科技股份有限公司 用于基因病症的核酸疗法
US12397012B2 (en) 2020-07-17 2025-08-26 Greenlight Biosciences, Inc. Nucleic acid therapeutics for genetic disorders

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Publication number Publication date
EP1549755A4 (en) 2006-09-20
EP1549755A2 (en) 2005-07-06
US20040180429A1 (en) 2004-09-16
WO2003104416A3 (en) 2004-07-01
AU2003247505A1 (en) 2003-12-22
WO2003104416A2 (en) 2003-12-18
CA2488249A1 (en) 2003-12-18

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