CA2488249A1 - Spliceosome mediated rna trans-splicing in stem cells - Google Patents

Spliceosome mediated rna trans-splicing in stem cells Download PDF

Info

Publication number
CA2488249A1
CA2488249A1 CA002488249A CA2488249A CA2488249A1 CA 2488249 A1 CA2488249 A1 CA 2488249A1 CA 002488249 A CA002488249 A CA 002488249A CA 2488249 A CA2488249 A CA 2488249A CA 2488249 A1 CA2488249 A1 CA 2488249A1
Authority
CA
Canada
Prior art keywords
stem cell
nucleic acid
acid molecule
mrna
target binding
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.)
Abandoned
Application number
CA002488249A
Other languages
English (en)
French (fr)
Inventor
Lloyd G. Mitchell
John Englehardt
Xiao Ming Liu
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Iowa Research Foundation UIRF
VirxSys Corp
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2488249A1 publication Critical patent/CA2488249A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • 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)
CA002488249A 2002-06-05 2003-06-05 Spliceosome mediated rna trans-splicing in stem cells Abandoned CA2488249A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US38659402P 2002-06-05 2002-06-05
US60/386,594 2002-06-05
PCT/US2003/017954 WO2003104416A2 (en) 2002-06-05 2003-06-05 Spliceosome mediated rna trans-splicing in stem cells

Publications (1)

Publication Number Publication Date
CA2488249A1 true CA2488249A1 (en) 2003-12-18

Family

ID=29736184

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002488249A Abandoned CA2488249A1 (en) 2002-06-05 2003-06-05 Spliceosome mediated rna trans-splicing in stem cells

Country Status (6)

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

Families Citing this family (7)

* 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
WO2022016077A1 (en) * 2020-07-17 2022-01-20 Greenlight Biosciences, Inc. Nucleic acid therapeutics for genetic disorders
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

Also Published As

Publication number Publication date
EP1549755A4 (en) 2006-09-20
EP1549755A2 (en) 2005-07-06
JP2005528912A (ja) 2005-09-29
US20040180429A1 (en) 2004-09-16
WO2003104416A3 (en) 2004-07-01
AU2003247505A1 (en) 2003-12-22
WO2003104416A2 (en) 2003-12-18

Similar Documents

Publication Publication Date Title
JP3343357B2 (ja) レトロウイルスによる標的細胞への遺伝子導入方法
DE69535560T2 (de) Ein verstärkter, durch viren vermittelter dna-transfer
US6670177B2 (en) Methods for enhanced virus-mediated DNA transfer using molecules with virus-and cell-binding domains
EP0667791A4 (en) ISOLATION, GROWTH, DIFFERENTIATION AND GENE TECHNOLOGICAL MANIPULATION OF HUMAN MUSCLE CELLS.
JP2021519587A (ja) マイクロrna発現構築物及びその使用
EP4255502A2 (en) Compositions and methods for the targeting of bcl11a
EP0457856A1 (en) Isolation growth and differentiation of human muscle cells
US20030143742A1 (en) Compositions for receptor/liposome mediated transfection and methods of using same
US20040180429A1 (en) Spliceosome mediated RNA trans-splicing in stem cells
Piersanti et al. Lentiviral transduction of human postnatal skeletal (stromal, mesenchymal) stem cells: in vivo transplantation and gene silencing
US6033907A (en) Enhanced virus-mediated DNA transfer
CN109072220A (zh) 由成纤维细胞直接制造心脏祖细胞和心肌细胞的方法
EP1430115A1 (en) Immortilized stem cells
KR20230125806A (ko) 선천성 근이영양증의 치료를 위한 치료용 lama2 페이로드
Zhang et al. Mesenchymal stem cells
DiGirolamo The potential of human bone marrow stromal cells as vehicles for ex vivo gene therapy applications
Scarpa et al. Advances toward gene therapy
Spampinato et al. Transfer and expression of DNA constructs into cells by using retrovirus vectors: A new tool for pharmacological research
HK40043609A (en) Micro rna expression constructs and uses thereof
HK1107581A (en) Enhanced virus-mediated dna transfer
MXPA96004240A (en) Improved transfer of dna mediated by vi
JPWO1996028560A1 (ja) 新規細胞株
HK1008049B (en) Transduced fibroblasts and use therefor

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued