JP7229467B2 - トランス-スプライシングRNA(tsRNA) - Google Patents

トランス-スプライシングRNA(tsRNA) Download PDF

Info

Publication number
JP7229467B2
JP7229467B2 JP2018551404A JP2018551404A JP7229467B2 JP 7229467 B2 JP7229467 B2 JP 7229467B2 JP 2018551404 A JP2018551404 A JP 2018551404A JP 2018551404 A JP2018551404 A JP 2018551404A JP 7229467 B2 JP7229467 B2 JP 7229467B2
Authority
JP
Japan
Prior art keywords
trans
splicing
tsrna
vector
cell
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.)
Active
Application number
JP2018551404A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019509756A (ja
JP2019509756A5 (https=
Inventor
パツェル・フォルカー
ポダー・スシュミタ
Original Assignee
ナショナル ユニバーシティ オブ シンガポール
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
Application filed by ナショナル ユニバーシティ オブ シンガポール filed Critical ナショナル ユニバーシティ オブ シンガポール
Publication of JP2019509756A publication Critical patent/JP2019509756A/ja
Publication of JP2019509756A5 publication Critical patent/JP2019509756A5/ja
Priority to JP2023011523A priority Critical patent/JP7570125B2/ja
Application granted granted Critical
Publication of JP7229467B2 publication Critical patent/JP7229467B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/14Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • A61P31/06Antibacterial agents for tuberculosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/16Antivirals for RNA viruses for influenza or rhinoviruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • A61P31/22Antivirals for DNA viruses for herpes viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Oncology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Communicable Diseases (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pulmonology (AREA)
  • Cell Biology (AREA)
JP2018551404A 2016-04-01 2017-03-31 トランス-スプライシングRNA(tsRNA) Active JP7229467B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023011523A JP7570125B2 (ja) 2016-04-01 2023-01-30 トランス-スプライシングRNA(tsRNA)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201605586 2016-04-01
GB1605586.5 2016-04-01
PCT/SG2017/050183 WO2017171654A1 (en) 2016-04-01 2017-03-31 Trans-splicing rna (tsrna)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023011523A Division JP7570125B2 (ja) 2016-04-01 2023-01-30 トランス-スプライシングRNA(tsRNA)

Publications (3)

Publication Number Publication Date
JP2019509756A JP2019509756A (ja) 2019-04-11
JP2019509756A5 JP2019509756A5 (https=) 2020-05-14
JP7229467B2 true JP7229467B2 (ja) 2023-02-28

Family

ID=59966148

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018551404A Active JP7229467B2 (ja) 2016-04-01 2017-03-31 トランス-スプライシングRNA(tsRNA)
JP2023011523A Active JP7570125B2 (ja) 2016-04-01 2023-01-30 トランス-スプライシングRNA(tsRNA)

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023011523A Active JP7570125B2 (ja) 2016-04-01 2023-01-30 トランス-スプライシングRNA(tsRNA)

Country Status (7)

Country Link
US (2) US11517583B2 (https=)
EP (1) EP3436590A4 (https=)
JP (2) JP7229467B2 (https=)
CN (1) CN109642241B (https=)
AU (1) AU2017240703B2 (https=)
SG (1) SG11201808538QA (https=)
WO (1) WO2017171654A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021076656A1 (en) * 2019-10-15 2021-04-22 University Of Massachusetts Rna editor-enhanced rna trans-splicing
EP4100533A1 (en) * 2020-02-07 2022-12-14 University of Rochester Ribozyme-mediated rna assembly and expression
GB202102118D0 (en) 2021-02-15 2021-03-31 Cambridge Entpr Ltd RNA Trans-Splicing molecule
WO2022220968A1 (en) * 2021-04-15 2022-10-20 U1 Bio, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells
GB202108855D0 (en) 2021-06-21 2021-08-04 Cambridge Entpr Ltd Methods of eukaryotic gene expression
CN113584169B (zh) * 2021-07-28 2024-06-18 南京大学 一种与肝癌相关的血清tsRNA标志物、探针及其应用
AU2022337146A1 (en) * 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
EP4466354A2 (en) * 2022-01-18 2024-11-27 National University of Singapore Trans-splicing rna (tsrna)
EP4522758A4 (en) 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
WO2025085691A1 (en) * 2023-10-18 2025-04-24 Tacit Therapeutics, Inc. Trans-splicing nucleic acid molecules and methods of use
CN118813791A (zh) * 2024-09-14 2024-10-22 中国人民解放军总医院第二医学中心 用于诊断寒冷暴露致心肌损伤的生物标志物、试剂盒及应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050202448A1 (en) 2004-03-15 2005-09-15 Digigenomics Co., Ltd. Methods for identification, assessment, prevention, and therapy of cancer
JP2015534811A (ja) 2012-11-02 2015-12-07 バウアー,ヨハン 癌の治療に使用されるrnaトランススプライシング分子(rtm)

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
ATE354957T1 (de) * 1995-12-15 2006-03-15 Intronn Inc Durch trans-spaltung erhaltene therapeutische molekule
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
MXPA04010410A (es) * 2002-04-30 2005-07-05 Icon Genetics Ag Vectores de amplificacion basados en trans-empalme.
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
EP1579004B1 (en) * 2002-10-23 2010-06-16 VIRxSYS Corporation Screening methods for identification of efficient pre-trans-splicing molecules
KR100958293B1 (ko) * 2008-03-27 2010-05-19 단국대학교 산학협력단 테오필린에 의해 표적 특이적 rna 치환 활성이 조절되는알로스테릭 트랜스―스플라이싱 그룹 i 리보자임
KR20110036996A (ko) 2009-10-05 2011-04-13 전남대학교산학협력단 편평세포암 진단 마커인 ho-1 및 이의 용도
WO2012056441A1 (en) * 2010-10-28 2012-05-03 Nanodoc Ltd. Compositions and methods for specific cleavage of exogenous rna in a cell
SG2014012207A (en) * 2011-08-12 2014-05-29 Virxsys Corp Compositions and methods for inducing apoptosis
KR101293620B1 (ko) 2011-08-19 2013-08-13 국립암센터 트랜스-스플라이싱 라이보자임 및 암치료 유전자를 포함하는 재조합 아데노바이러스 및 이의 용도
KR101429696B1 (ko) * 2012-11-21 2014-08-13 국립암센터 안전성 및 항암활성이 증가된 재조합 아데노바이러스 및 이의 용도
US20140283156A1 (en) * 2013-03-14 2014-09-18 Cold Spring Harbor Laboratory Trans-splicing ribozymes and silent recombinases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050202448A1 (en) 2004-03-15 2005-09-15 Digigenomics Co., Ltd. Methods for identification, assessment, prevention, and therapy of cancer
JP2015534811A (ja) 2012-11-02 2015-12-07 バウアー,ヨハン 癌の治療に使用されるrnaトランススプライシング分子(rtm)

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BMC Cancer,2013年,Vol.13, article No.137,p.1-10
Molecular Oncology,2013年,Vol.7,p.1056-1068
SUSHMITA PODDAR,Chapter4 Results Trans-splicing RNA molecules for 3’and 5’exon labelling (EL) targeting alpha-fetoprotein (AFP), Chapter5 Results Selective killing of AFP positive cells triggered by trans-splicing,RNA trans-splicing for selective killing of virus-transduced or cancer cells,2016年01月,p.67-141
SUSHMITA PODDAR,Chapter4 Results Trans-splicing RNA molecules for 3’and 5’exon labelling (EL) targeting alpha-fetoprotein (AFP),RNA trans-splicing for selective killing of virus-transduced or cancer cells,2016年01月,p.67-110

Also Published As

Publication number Publication date
US20240075051A1 (en) 2024-03-07
CN109642241B (zh) 2023-06-27
JP7570125B2 (ja) 2024-10-21
WO2017171654A1 (en) 2017-10-05
CN109642241A (zh) 2019-04-16
US11517583B2 (en) 2022-12-06
JP2019509756A (ja) 2019-04-11
SG11201808538QA (en) 2018-10-30
US20190111072A1 (en) 2019-04-18
JP2023055809A (ja) 2023-04-18
EP3436590A4 (en) 2019-12-04
AU2017240703B2 (en) 2020-05-21
EP3436590A1 (en) 2019-02-06
AU2017240703A1 (en) 2018-10-18

Similar Documents

Publication Publication Date Title
JP7570125B2 (ja) トランス-スプライシングRNA(tsRNA)
EP3230460B2 (en) Methods and compositions for selectively eliminating cells of interest
EP2871239B1 (en) Cell capable of producing adeno-associated virus vector
US11939575B2 (en) Modified tracrRNAs gRNAs, and uses thereof
CN113302291B (zh) 使用逆转录病毒整合酶-Cas9融合蛋白通过定向非同源DNA插入进行的基因组编辑
JP2017506893A (ja) ウイルス複製不活化組成物並びにその製造方法及び使用
CN110177578A (zh) modRNA的细胞特异性表达
Fechner et al. Coxsackievirus B3 and adenovirus infections of cardiac cells are efficiently inhibited by vector-mediated RNA interference targeting their common receptor
JP2007503205A (ja) 多コンパートメント真核生物発現系
KR101478869B1 (ko) 마이크로rna를 이용한 조절을 통한 암 특이적 유전자 치료제
JP2025156372A (ja) 免疫療法に対する腫瘍細胞の感作のための方法及び組成物
EP4491720A1 (en) Compositions and methods for increasing deletion efficiency of nucleic acid segment by modulation of nhej repair pathway
US20250145990A1 (en) Trans-splicing rna (tsrna)
KR20260032655A (ko) Hbv 유전자 발현의 후성유전학적 조절을 위한 조성물 및 방법
Cordelier et al. Replication-deficient rSV40 mediate pancreatic gene transfer and long-term inhibition of tumor growth
JP6483019B2 (ja) 新規アデノウイルス及びその増殖促進方法
CN118556124A (zh) 经工程化的casx阻遏物系统
KR20220018410A (ko) 세포질에서 자가전사가 가능하고 유전체 편집을 제공하는 rna/dna 시스템
JP4505566B2 (ja) 肺癌治療剤
KR20250158061A (ko) Pah-조절 시스템 및 방법
KR20230127070A (ko) 세포질에서 자가 전사가 가능하고 발현억제 rna를 제공하는 rna/dna 시스템
She Design, investigation and delivery of advanced trans-splicing RNA for suicide gene therapy
CN120882872A (zh) 核酶介导的rna组装和表达
HK40109408A (zh) 一种环状rna、组合物和治疗疾病的方法
HK1244841B (en) Methods and compositions for selectively eliminating cells of interest

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190903

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200330

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20200330

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200330

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210512

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210721

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210924

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211104

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220330

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220621

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: 20221214

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230106

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230131

R150 Certificate of patent or registration of utility model

Ref document number: 7229467

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250