JP4353390B2 - スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物 - Google Patents

スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物 Download PDF

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JP4353390B2
JP4353390B2 JP2000565168A JP2000565168A JP4353390B2 JP 4353390 B2 JP4353390 B2 JP 4353390B2 JP 2000565168 A JP2000565168 A JP 2000565168A JP 2000565168 A JP2000565168 A JP 2000565168A JP 4353390 B2 JP4353390 B2 JP 4353390B2
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splicing
trans
mrna
ptm
cell
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JP2004504001A (ja
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ミッチェル、ロイド、ジー
− ブランコ、マリアーノ、エイ ガルシア
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バークシス コーポレイション
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    • C12N2840/445Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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JP2000565168A 1998-08-13 1999-08-12 スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物 Expired - Fee Related JP4353390B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/133,717 US6083702A (en) 1995-12-15 1998-08-13 Methods and compositions for use in spliceosome mediated RNA trans-splicing
US09/158,863 US6280978B1 (en) 1995-12-15 1998-09-23 Methods and compositions for use in spliceosome mediated RNA trans-splicing
PCT/US1999/018371 WO2000009734A2 (en) 1998-08-13 1999-08-12 Methods and compositions for use in spliceosome mediated rna trans-splicing

Related Child Applications (2)

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JP2007177115A Division JP2007312780A (ja) 1998-08-13 2007-07-05 スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物
JP2008182795A Division JP2009000120A (ja) 1998-08-13 2008-07-14 スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物

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JP2004504001A JP2004504001A (ja) 2004-02-12
JP2004504001A5 JP2004504001A5 (enExample) 2005-02-17
JP4353390B2 true JP4353390B2 (ja) 2009-10-28

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JP2007177115A Withdrawn JP2007312780A (ja) 1998-08-13 2007-07-05 スプライソソーム媒介rnaトランス−スプライシングにおける使用のための方法及び組成物

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US (1) US6280978B1 (enExample)
EP (2) EP1105513A2 (enExample)
JP (2) JP4353390B2 (enExample)
AU (1) AU773186B2 (enExample)
CA (1) CA2339300C (enExample)
IL (1) IL141409A0 (enExample)
WO (1) WO2000009734A2 (enExample)

Families Citing this family (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020115207A1 (en) * 1995-12-15 2002-08-22 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20030027250A1 (en) * 1995-12-15 2003-02-06 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20060088938A1 (en) * 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US20030077754A1 (en) * 1995-12-15 2003-04-24 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
FR2790955B1 (fr) * 1999-03-19 2003-01-17 Assist Publ Hopitaux De Paris Utilisation d'oligonucleotides stabilises comme principe actif antitumoral
GB2362383B (en) * 2000-05-19 2003-12-31 Devgen Nv Gene expression system
WO2002008408A2 (en) * 2000-07-21 2002-01-31 Trustees Of Boston University Modular vector systems
DE10057014B8 (de) * 2000-11-17 2008-04-30 Steven Dooley Verfahren zur Herstellung von zielzellspezifischen Arzneimitteln
EP1358203A4 (en) * 2001-01-08 2006-11-22 Intronn Inc METHOD AND COMPOSITION FOR CONNECTING IN SPLICEOSOME-MEDIATED RNA TRANS SPLICING
US20040126774A1 (en) * 2001-01-08 2004-07-01 Mitchell Lioyd G. Correction of factor VIII genetic defects using spliceosome mediated RNA trans splicing
DE10139492B4 (de) * 2001-08-13 2004-06-09 Eul, Joachim, Dr. Verfahren zur Reparatur einer mutierten RNA aus einer gendefekten DNA und zum gezielten Abtöten von Tumorzellen durch RNA-Transspleißen sowie Verfahren zum Nachweis von natürlich-transgespleißter zellulärer RNA
US20030153054A1 (en) * 2002-02-12 2003-08-14 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
CA2477295A1 (en) * 2002-02-25 2003-09-04 Intronn, Inc. Trans-splicing mediated imaging of gene expression
US7399753B2 (en) * 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
US20040214263A1 (en) * 2002-03-20 2004-10-28 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing
US20040018520A1 (en) * 2002-04-22 2004-01-29 James Thompson Trans-splicing enzymatic nucleic acid mediated biopharmaceutical and protein
US20030204861A1 (en) * 2002-04-30 2003-10-30 Madaiah Puttaraju Transgenic animal model for spliceosome-mediated RNA trans-splicing
JP2005536231A (ja) * 2002-05-08 2005-12-02 イントロン,インコーポレーテッド アデノウイルスに細胞選択的複製をもたらすための、スプライセオソーム媒介型rnaトランススプライシングの使用
WO2003104412A2 (en) 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing and correction of factor viii genetic defects using spliceosome mediated rna trans spling
EP1549755A4 (en) * 2002-06-05 2006-09-20 Intronn Inc SPREAD OSOM-MEDIATED TRANS-RNA SPLEISSEN IN STEM CELLS
US20040018622A1 (en) * 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
AU2003285009B2 (en) * 2002-10-23 2010-10-07 Virxsys Corporation Screening methods for identification of efficient pre-trans-splicing molecules
WO2004087958A1 (en) * 2003-03-31 2004-10-14 Council Of Scientific And Industrial Research Stn gene oligonucleotide primers for detecting salmonella species and detection prosses using the same
US8053232B2 (en) * 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
CA2553828A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. Expression of apoa-1 and variants thereof using spliceosome mediated rna trans-splicing
US20060094110A1 (en) * 2004-07-30 2006-05-04 Mcgarrity Gerard J Use of spliceosome mediated RNA trans-splicing for immunotherapy
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
DE102004047492B4 (de) * 2004-09-23 2006-07-20 Jost-Werke Gmbh & Co. Kg Verfahren zum Übertragen von elektrischer, pneumatischer oder hydraulischer Energie sowie ein Energieübertragungssystem
US7871795B2 (en) * 2004-10-08 2011-01-18 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
JP5017118B2 (ja) * 2004-10-08 2012-09-05 バークシス コーポレーション 組換えタンパク質をインビボ生産するための、非常に豊富な転写産物の標的化トランススプライシング
US7879321B2 (en) * 2004-10-08 2011-02-01 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
DE102006009000B3 (de) * 2006-02-23 2007-09-06 Tutech Innovation Gmbh Rechengen
JP5081462B2 (ja) * 2007-02-02 2012-11-28 富士フイルム株式会社 トランススプライシング法による融合タンパク質作製方法
EP2151248A1 (en) * 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
EP2326711A2 (en) * 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
US8923125B2 (en) * 2008-10-24 2014-12-30 Qualcomm Incorporated Wireless network resource adaptation
ES2578993T3 (es) 2009-10-08 2016-08-03 Association Institut De Myologie Moléculas de ácido nucleico y métodos para intercambiar uno o más exones mediante corte y empalme en trans
WO2013016595A1 (en) 2011-07-28 2013-01-31 The Regents Of The University Of California Exonic splicing enhancers and exonic splicing silencers
WO2014045283A1 (en) * 2012-09-24 2014-03-27 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
US9447411B2 (en) 2013-01-14 2016-09-20 Cellecta, Inc. Methods and compositions for single cell expression profiling
WO2017087900A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
CN109642241B (zh) * 2016-04-01 2023-06-27 新加坡国立大学 反式剪接RNA(tsRNA)
KR20250150157A (ko) 2018-04-17 2025-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
EP4013873A1 (en) 2019-08-16 2022-06-22 Massachusetts Institute Of Technology Targeted trans-splicing using crispr/cas13
EP4522758A2 (en) 2022-05-13 2025-03-19 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules
EP4590825A1 (en) * 2022-09-22 2025-07-30 Rna Morph Ltd Folding oligonucleotides
EP4594505A1 (en) 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
CN120641122A (zh) 2022-11-23 2025-09-12 安铂生物公司 基因修饰核酸内切酶
WO2025128343A1 (en) 2023-12-11 2025-06-19 Just-Evotec Biologics, Inc. Protein expression using trans-splicing and split selectable markers

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ241310A (en) * 1991-01-17 1995-03-28 Gen Hospital Corp Trans-splicing ribozymes
NZ241311A (en) * 1991-01-17 1995-03-28 Gen Hospital Corp Rna sequence having trans-splicing activity, plant strains
US5498531A (en) * 1993-09-10 1996-03-12 President And Fellows Of Harvard College Intron-mediated recombinant techniques and reagents
US5667969A (en) * 1993-11-12 1997-09-16 University Research Corporation Alteration of sequence of a deleterious target molecule by ribozyme catalyzed trans-splicing
EP0883344B2 (en) * 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing

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