JP2004504001A5 - - Google Patents
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- Publication number
- JP2004504001A5 JP2004504001A5 JP2000565168A JP2000565168A JP2004504001A5 JP 2004504001 A5 JP2004504001 A5 JP 2004504001A5 JP 2000565168 A JP2000565168 A JP 2000565168A JP 2000565168 A JP2000565168 A JP 2000565168A JP 2004504001 A5 JP2004504001 A5 JP 2004504001A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- cell
- target
- mrna
- 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.)
- Granted
Links
- 108020004707 nucleic acids Proteins 0.000 claims 110
- 102000039446 nucleic acids Human genes 0.000 claims 110
- 150000007523 nucleic acids Chemical class 0.000 claims 110
- 230000027455 binding Effects 0.000 claims 104
- 238000009739 binding Methods 0.000 claims 104
- 108020004999 messenger RNA Proteins 0.000 claims 92
- 239000002773 nucleotide Substances 0.000 claims 63
- 125000003729 nucleotide group Chemical group 0.000 claims 63
- 108020005067 RNA Splice Sites Proteins 0.000 claims 48
- 125000006850 spacer group Chemical group 0.000 claims 27
- 239000013598 vector Substances 0.000 claims 23
- 108090000765 processed proteins & peptides Proteins 0.000 claims 17
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 claims 16
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 16
- 229920001184 polypeptide Polymers 0.000 claims 15
- 102000004196 processed proteins & peptides Human genes 0.000 claims 15
- 108091092236 Chimeric RNA Proteins 0.000 claims 13
- 238000000034 method Methods 0.000 claims 13
- 125000000714 pyrimidinyl group Chemical group 0.000 claims 11
- 230000000295 complement effect Effects 0.000 claims 8
- 239000013604 expression vector Substances 0.000 claims 6
- 230000003993 interaction Effects 0.000 claims 6
- 230000001404 mediated effect Effects 0.000 claims 6
- 201000003883 Cystic fibrosis Diseases 0.000 claims 5
- 230000015572 biosynthetic process Effects 0.000 claims 5
- 238000004519 manufacturing process Methods 0.000 claims 5
- 230000004927 fusion Effects 0.000 claims 4
- 102000034356 gene-regulatory proteins Human genes 0.000 claims 2
- 108091006104 gene-regulatory proteins Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 238000003259 recombinant expression Methods 0.000 claims 2
- 102000005720 Glutathione transferase Human genes 0.000 claims 1
- 108010070675 Glutathione transferase Proteins 0.000 claims 1
- 108020001507 fusion proteins Proteins 0.000 claims 1
- 102000037865 fusion proteins Human genes 0.000 claims 1
- 239000003550 marker Substances 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
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)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2004504001A JP2004504001A (ja) | 2004-02-12 |
| JP2004504001A5 true JP2004504001A5 (https=) | 2005-02-17 |
| JP4353390B2 JP4353390B2 (ja) | 2009-10-28 |
Family
ID=26831623
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2000565168A Expired - Fee Related JP4353390B2 (ja) | 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 |
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US6280978B1 (https=) |
| EP (2) | EP1489185A1 (https=) |
| JP (2) | JP4353390B2 (https=) |
| AU (1) | AU773186B2 (https=) |
| CA (1) | CA2339300C (https=) |
| IL (1) | IL141409A0 (https=) |
| WO (1) | WO2000009734A2 (https=) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060088938A1 (en) * | 1995-12-15 | 2006-04-27 | Mitchell Lloyd G | Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants |
| US20020193580A1 (en) * | 1995-12-15 | 2002-12-19 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US20020115207A1 (en) * | 1995-12-15 | 2002-08-22 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US20030077754A1 (en) * | 1995-12-15 | 2003-04-24 | 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 |
| 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 |
| AU2001277014A1 (en) * | 2000-07-21 | 2002-02-05 | Trustees Of Boston University | Modular vector systems |
| DE10057014B8 (de) * | 2000-11-17 | 2008-04-30 | Steven Dooley | Verfahren zur Herstellung von zielzellspezifischen Arzneimitteln |
| JP2004525618A (ja) * | 2001-01-08 | 2004-08-26 | イントロン，インコーポレーテッド | スプライセオソームにより媒介されるｒｎａトランススプライシング |
| 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 |
| EP1521766B1 (en) | 2002-06-05 | 2012-11-07 | VIRxSYS Corporation | SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING |
| AU2003247505A1 (en) * | 2002-06-05 | 2003-12-22 | University Of Iowa Research Foundation | Spliceosome mediated rna trans-splicing in stem cells |
| US20040018622A1 (en) * | 2002-07-17 | 2004-01-29 | Mitchell Lloyd G. | Spliceosome mediated RNA trans-splicing for correction of skin disorders |
| EP1579004B1 (en) * | 2002-10-23 | 2010-06-16 | VIRxSYS Corporation | Screening methods for identification of efficient pre-trans-splicing molecules |
| AU2003226642A1 (en) * | 2003-03-31 | 2004-10-25 | 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 |
| EP1716165A4 (en) * | 2004-01-23 | 2008-06-18 | Virxsys Corp | EXPRESSION OF APOA-1 AND VARIANTS THEREOF, USING SPSSISSOSOM-MEDIATED RNA TRANS MIXING |
| US7968334B2 (en) * | 2004-01-23 | 2011-06-28 | Virxsys Corporation | Expression of apoAI 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 |
| EP1831365B1 (en) * | 2004-10-08 | 2013-08-07 | 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 |
| WO2010022194A2 (en) * | 2008-08-20 | 2010-02-25 | 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 |
| US9303078B2 (en) | 2009-10-08 | 2016-04-05 | Association Institut De Myologie | Nucleic acid molecules and methods for exchanging exon(s) by transsplicing |
| 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 |
| WO2014110509A1 (en) * | 2013-01-14 | 2014-07-17 | Cellecta, Inc. | Methods and compositions for single cell expression profiling |
| CA3005474A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| EP3436590A4 (en) | 2016-04-01 | 2019-12-04 | National University of Singapore | TRANSSPLEISSEN OF RNA (TSRNA) |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| CN115023499A (zh) | 2019-08-16 | 2022-09-06 | 麻省理工学院 | 使用CRISPR/Cas13的靶向反式剪接 |
| EP4522758A4 (en) | 2022-05-13 | 2026-05-06 | Ascidian Therapeutics Inc | Abca4 trans-splicing molecules |
| WO2024062487A1 (en) * | 2022-09-22 | 2024-03-28 | 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 |
| WO2024112957A1 (en) | 2022-11-23 | 2024-05-30 | Amber Bio Inc. | Gene-modifying endonucleases |
| 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ314630A (en) * | 1991-01-17 | 2000-11-24 | Harvard College | Use of trans-splicing ribozymes for genetic modification and cell ablation in a host cell |
| NZ314629A (en) * | 1991-01-17 | 2000-08-25 | Gen Hospital Corp | Use trans-splicing ribozymes to prepare medicaments for gene therapies |
| 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 |
| ATE354957T1 (de) * | 1995-12-15 | 2006-03-15 | Intronn Inc | Durch trans-spaltung erhaltene therapeutische molekule |
-
1998
- 1998-09-23 US US09/158,863 patent/US6280978B1/en not_active Expired - Lifetime
-
1999
- 1999-08-12 WO PCT/US1999/018371 patent/WO2000009734A2/en not_active Ceased
- 1999-08-12 AU AU56735/99A patent/AU773186B2/en not_active Ceased
- 1999-08-12 EP EP04077408A patent/EP1489185A1/en not_active Withdrawn
- 1999-08-12 IL IL14140999A patent/IL141409A0/xx unknown
- 1999-08-12 EP EP99943690A patent/EP1105513A2/en not_active Withdrawn
- 1999-08-12 JP JP2000565168A patent/JP4353390B2/ja not_active Expired - Fee Related
- 1999-08-12 CA CA2339300A patent/CA2339300C/en not_active Expired - Fee Related
-
2007
- 2007-07-05 JP JP2007177115A patent/JP2007312780A/ja not_active Withdrawn
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