JP2004504001A - スプライソソーム媒介ｒｎａトランスー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トランスー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

Info

Publication number
JP2004504001A
JP2004504001A JP2000565168A JP2000565168A JP2004504001A JP 2004504001 A JP2004504001 A JP 2004504001A JP 2000565168 A JP2000565168 A JP 2000565168A JP 2000565168 A JP2000565168 A JP 2000565168A JP 2004504001 A JP2004504001 A JP 2004504001A
Authority
JP
Japan
Prior art keywords
trans
mrna
splicing
cells
present
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
Application number
JP2000565168A
Other languages
English (en)
Other versions
JP4353390B2 (ja
JP2004504001A5 (ja
Inventor
ミッチェル、ロイド、ジー
ガルシア　−　ブランコ、マリアーノ、エイ
Original Assignee
イントロン　ホールディングス、エルエルシー
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
Priority claimed from US09/133,717 external-priority patent/US6083702A/en
Application filed by イントロン　ホールディングス、エルエルシー filed Critical イントロン　ホールディングス、エルエルシー
Publication of JP2004504001A publication Critical patent/JP2004504001A/ja
Publication of JP2004504001A5 publication Critical patent/JP2004504001A5/ja
Application granted granted Critical
Publication of JP4353390B2 publication Critical patent/JP4353390B2/ja
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Images

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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1027—Mutagenizing nucleic acids by DNA shuffling, e.g. RSR, STEP, RPR
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/34—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Corynebacterium (G)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/59—Follicle-stimulating hormone [FSH]; Chorionic gonadotropins, e.g. HCG; Luteinising hormone [LH]; Thyroid-stimulating hormone [TSH]
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • C12N15/63—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors 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

Abstract

本発明の分子及び方法は、選択された細胞のサブセット中で、トランス−スプライスされた分子のインビボでの製造のための手段を提供する。本発明の前−トランス−スプライシング分子は、前−トランス−スプライシング分子と特定の標的細胞において唯一発現される、前−ｍＲＮＡとの間のトランス−スプライシング反応のための基質である。インビボでのトランス−スプライシング反応は、ｍＲＮＡとして機能的な又は、ターゲット細胞において発現されるべきタンパクをコードする。ｍＲＮＡの発現生成物は、細胞又は宿主生物に対して治療上の価値を持つタンパクであるか、特定の細胞の死を誘発する毒素か、またはそのような細胞に通常存在しないタンパクである。本発明はさらに、エキソンタグ方法を用いて前−ｍＲＮＡのエキソン／イントロンの境界を同定するための、遺伝子操作されたＰＴＭｓを提供する。本発明のＰＴＭｓは、特定の細胞タイプにおいて発現されるタンパクの精製及び同定に用いることができる、ペプチドアフィニティ精製タグをコードするキメラＲＮＡの製造を生じる。

Description

Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001
Figure 2004504001

Claims (15)

  1. Figure 2004504001
  2. Figure 2004504001
  3. Figure 2004504001
  4. Figure 2004504001
  5. Figure 2004504001
  6. Figure 2004504001
  7. Figure 2004504001
  8. Figure 2004504001
  9. Figure 2004504001
  10. Figure 2004504001
  11. Figure 2004504001
  12. Figure 2004504001
  13. Figure 2004504001
  14. Figure 2004504001
  15. Figure 2004504001
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)

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 true JP2004504001A (ja) 2004-02-12
JP2004504001A5 JP2004504001A5 (ja) 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 (ja)
EP (2) EP1105513A2 (ja)
JP (2) JP4353390B2 (ja)
AU (1) AU773186B2 (ja)
CA (1) CA2339300C (ja)
IL (1) IL141409A0 (ja)
WO (1) WO2000009734A2 (ja)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008515435A (ja) * 2004-10-08 2008-05-15 バークシス コーポレーション 抗体遺伝子導入および抗体ポリペプチド産生のためのｒｎａトランススプライシングの使用
JP2008515441A (ja) * 2004-10-08 2008-05-15 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
JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030027250A1 (en) * 1995-12-15 2003-02-06 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
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
US20060088938A1 (en) * 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
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
AU2002246959B2 (en) * 2001-01-08 2008-03-06 Virxsys Corporation 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)
AU2003213270A1 (en) * 2002-02-25 2003-09-09 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
CA2485341A1 (en) * 2002-05-08 2004-06-17 Intronn, Inc. Use of spliceosome mediated rna trans-splicing to confer cell selective replication to adenoviruses
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
EP1549755A4 (en) * 2002-06-05 2006-09-20 Intronn Inc Reaction of RNA by EPISSEOME IN STEM CELLS
US20040018622A1 (en) * 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
WO2004038380A2 (en) * 2002-10-23 2004-05-06 Intronn, Inc. 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
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
JP2007518423A (ja) * 2004-01-23 2007-07-12 イントロン、インコーポレイテッド スプライセオソーム仲介型ｒｎａトランススプライシングを使用するアポａ−１及びその変異体の発現
WO2005070948A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects 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
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
US20110263015A1 (en) * 2008-08-20 2011-10-27 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
WO2011042556A1 (en) 2009-10-08 2011-04-14 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
AU2013319788B2 (en) * 2012-09-24 2019-04-11 Yissum Reasearch 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
WO2017087900A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
AU2017240703B2 (en) * 2016-04-01 2020-05-21 National University Of Singapore Trans-splicing RNA (tsRNA)
KR20220044811A (ko) 2019-08-16 2022-04-11 메사추세츠 인스티튜트 오브 테크놀로지 Crispr/cas13을 사용하는 표적화된 트랜스-이어맞추기
WO2024062487A1 (en) * 2022-09-22 2024-03-28 Rna Morph Ltd Folding oligonucleotides
WO2024068898A1 (en) 2022-09-30 2024-04-04 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

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
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
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
WO1997022250A1 (en) * 1995-12-15 1997-06-26 Intronn Llc Therapeutic molecules generated by trans-splicing

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008515435A (ja) * 2004-10-08 2008-05-15 バークシス コーポレーション 抗体遺伝子導入および抗体ポリペプチド産生のためのｒｎａトランススプライシングの使用
JP2008515441A (ja) * 2004-10-08 2008-05-15 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
JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用

Also Published As

Publication number Publication date
AU5673599A (en) 2000-03-06
WO2000009734A9 (en) 2000-07-13
WO2000009734A3 (en) 2000-05-18
CA2339300C (en) 2015-11-24
JP2007312780A (ja) 2007-12-06
JP4353390B2 (ja) 2009-10-28
EP1105513A2 (en) 2001-06-13
EP1489185A1 (en) 2004-12-22
AU773186B2 (en) 2004-05-20
US6280978B1 (en) 2001-08-28
CA2339300A1 (en) 2000-02-24
WO2000009734A2 (en) 2000-02-24
IL141409A0 (en) 2002-03-10

Similar Documents

Publication Publication Date Title
JP2004504001A (ja) スプライソソーム媒介ｒｎａトランスースプライシングにおける使用のための方法及び組成物
CN107488649A (zh) 一种Cpf1和p300核心结构域的融合蛋白、相应的DNA靶向激活系统和应用
ATE527368T1 (de) Axmi-010, ein delta-endotoxin-gen, und verfahren zur verwendung davon
BRPI0511118A (pt) polipeptìdeos de haemophilus influenzae não transcritos
EA200701023A1 (ru) Отбор клеток-хозяев, экспрессирующих белок на высоких уровнях
ATE548378T1 (de) Axmi-036 ein delta-endotoxin-gen und verfahren zur dessen verwendung
EA201071323A1 (ru) Axmi-115, axmi-113, axmi-005, axmi-163, axmi-184 инсектицидные белки и способы их применения
EP0994190A3 (en) DNA conferring L-homoserine resistance to bacteria, and its use
EP1954706A4 (en) SELECTIVE TERMINAL TAGGING OF NUCLEIC ACIDS
EA201070371A1 (ru) Axmi-066 и axmi-076: дельта-эндотоксиновые белки и способы их применения
WO2007147096A3 (en) A family of pesticidal proteins and methods for their use
WO2004083225A3 (fr) Biopuces a sondes et leurs methodes d’utilisation
HK1082761A1 (en) Circular dna molecule having a conditional origin of replication, process for their preparation and their use in gene therapy
WO2007040592A8 (en) Making nucleic acid sequences in parallel and use
DE69840387D1 (de) Nik proteine, nukleinsäure sowie verfahren
DE10345772A1 (de) Verfahren zur Herstellung von 3-Methylamino-1-(thien-2-yl)-propan-1-ol
DE69941089D1 (de) Transferverfahren für die spezifische zelluläre lokalisation von nukleinsäuren
Ueta et al. Ribosomal protein L31 in Escherichia coli contributes to ribosome subunit association and translation, whereas short L31 cleaved by protease 7 reduces both activities
MX341785B (es) Sistemas de sintasa de poliquetida pufa y uso de los mismos.
ATE474922T1 (de) Fluoreszenzproteine aus copepoda-spezies und verfahren zur verwendung davon
WO2002053581A3 (en) Spliceosome mediated rna trans-splicing
CN101268186A (zh) 新的内切核糖核酸酶
WO2002044409A3 (en) Substrate linked directed evolution (slide)
DE602006004966D1 (de) Intrinsic-faktor vom schwein
Nakai et al. Sequence‐specific cleavage of RNA by a hybrid ribonuclease H

Legal Events

Date Code Title Description
A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20051220

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20060320

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20060328

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20060620

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20070105

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20070330

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20070425

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20070705

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20071203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20080125

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20080314

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20080612

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20080804

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A132

Effective date: 20081107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20090209

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20090217

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20090309

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20090316

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20090407

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20090414

RD13 Notification of appointment of power of sub attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7433

Effective date: 20090422

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20090507

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20090423

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20090707

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

RD17 Notification of extinguishment of power of sub attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7437

Effective date: 20090710

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20090723

R150 Certificate of patent or registration of utility model

Free format text: JAPANESE INTERMEDIATE CODE: R150

FPAY Renewal fee payment (event date is renewal date of database)

Free format text: PAYMENT UNTIL: 20120807

Year of fee payment: 3

LAPS Cancellation because of no payment of annual fees