WO2011146885A3 - Compositions and methods for lentiviral expression of apoa-1 or variants thereof using spliceosome mediated rna trans-splicing - Google Patents

Compositions and methods for lentiviral expression of apoa-1 or variants thereof using spliceosome mediated rna trans-splicing Download PDF

Info

Publication number
WO2011146885A3
WO2011146885A3 PCT/US2011/037419 US2011037419W WO2011146885A3 WO 2011146885 A3 WO2011146885 A3 WO 2011146885A3 US 2011037419 W US2011037419 W US 2011037419W WO 2011146885 A3 WO2011146885 A3 WO 2011146885A3
Authority
WO
WIPO (PCT)
Prior art keywords
splicing
compositions
trans
target
apoa
Prior art date
Application number
PCT/US2011/037419
Other languages
French (fr)
Other versions
WO2011146885A2 (en
Inventor
Madaiah Puttaraju
Gary Stephen Mansfield
Nikolay Korokhov
Jenice G. D'costa
Laurent M. Humeau
Gerard J. Mcgarrity
Jun Wang
Original Assignee
Virxsys Corporation
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 Virxsys Corporation filed Critical Virxsys Corporation
Publication of WO2011146885A2 publication Critical patent/WO2011146885A2/en
Publication of WO2011146885A3 publication Critical patent/WO2011146885A3/en

Links

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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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

The disclosure provides methods and compositions for generating nucleic acid molecules through RNA trans-splicing that target a highly expressed pre-mRNA and contain the coding sequence of a protein or polypeptide of interest. The compositions disclosed include recombinant lentiviral vectors expressing pre-trans-splicing molecules (PTMs) designed to interact with the target precursor messenger RNA molecule (target premRNA) that is abundantly expressed, and mediate a trans-splicing reaction resulting in the generation of chimeric RNA molecule (chimeric RNA) capable of encoding a protein or polypeptide of interest.
PCT/US2011/037419 2010-05-21 2011-05-20 Compositions and methods for lentiviral expression of apoa-1 or variants thereof using spliceosome mediated rna trans-splicing WO2011146885A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US34734910P 2010-05-21 2010-05-21
US61/347,349 2010-05-21

Publications (2)

Publication Number Publication Date
WO2011146885A2 WO2011146885A2 (en) 2011-11-24
WO2011146885A3 true WO2011146885A3 (en) 2012-03-15

Family

ID=44992368

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/037419 WO2011146885A2 (en) 2010-05-21 2011-05-20 Compositions and methods for lentiviral expression of apoa-1 or variants thereof using spliceosome mediated rna trans-splicing

Country Status (1)

Country Link
WO (1) WO2011146885A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060029596A1 (en) * 1999-07-12 2006-02-09 Wold William S Replication-competent anti-cancer vectors
US20060172381A1 (en) * 2004-10-08 2006-08-03 Mcgarrity Gerard J Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
US20060294614A1 (en) * 2001-08-22 2006-12-28 Wyeth Murine genomic polynucleotide sequence encoding a G-protein coupled receptor and methods of use therefor
US20070196922A1 (en) * 2001-10-02 2007-08-23 Didier Trono Methods and Compositions Relating to Restricted Expression Lentiviral Vectors and Their Applications

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060029596A1 (en) * 1999-07-12 2006-02-09 Wold William S Replication-competent anti-cancer vectors
US20060294614A1 (en) * 2001-08-22 2006-12-28 Wyeth Murine genomic polynucleotide sequence encoding a G-protein coupled receptor and methods of use therefor
US20070196922A1 (en) * 2001-10-02 2007-08-23 Didier Trono Methods and Compositions Relating to Restricted Expression Lentiviral Vectors and Their Applications
US20060172381A1 (en) * 2004-10-08 2006-08-03 Mcgarrity Gerard J Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
WANG ET AL.: "Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo.", MOL THER, vol. 17, no. 2, February 2009 (2009-02-01), pages 343 - 351 *
YANG ET AL.: "Spliceosome-Mediated RNA Trans-splicing.", MOL THER., vol. 12, no. 6, 2005, pages 1006 - 1012 *

Also Published As

Publication number Publication date
WO2011146885A2 (en) 2011-11-24

Similar Documents

Publication Publication Date Title
WO2012045082A3 (en) Engineered nucleic acids and methods of use thereof
MY177065A (en) 4-1bb binding molecules
WO2010139736A3 (en) Recombinant production of peptides
WO2008145400A3 (en) Mutated structural protein of a parvovirus
WO2010120514A3 (en) Antigen-binding proteins comprising recombinant protein scaffolds
MX2010008099A (en) Stabilized angiopoietin-2 antibodies and uses thereof.
EP2274442A4 (en) Consensus sequences of chikungunya viral proteins, nucleic acid molecules encoding the same, and compositions and methods for using the same
MX2014007233A (en) Modified nucleoside, nucleotide, and nucleic acid compositions.
WO2013119302A3 (en) Gh61 polypeptide variants and polynucleotides encoding same
WO2012044915A3 (en) Beta-glucosidase variants and polynucleotides encoding same
WO2012065164A3 (en) Consensus prostate antigens nucleic acid molecule encoding the same and vaccine and uses comprising the same
WO2013040093A3 (en) Glucagon-like peptide-2 compositions and methods of making and using same
WO2013006953A8 (en) Genes and proteins for alkanoyl-coa synthesis
WO2009109597A8 (en) Method for producing alpha- santalene
WO2011113027A3 (en) Npp1 fusion proteins
WO2010020766A3 (en) Interleukin fusion polypeptides
WO2007095201A3 (en) Pseudotyped retroviral vectors and methods of use thereof
WO2010066411A3 (en) Polypeptides having cellobiohydrolase ii activity
WO2006083331A3 (en) Use of rna trans-splicing for antibody gene transfer and antibody polypeptide production
WO2013096603A3 (en) Cellobiohydrolase variants and polynucleotides encoding same
IL215014A (en) Antibody molecules having binding specificity for human il-13, compositions comprising them, dna sequences encoding them, cloning vectors and host cells, process for producing and uses thereof
WO2010088463A3 (en) Polypeptides having expansin activity and polynucleotides encoding same
WO2013074956A3 (en) Polypeptides having beta-glucosidase activity, beta-xylosidase activity, or beta-glucosidase and beta-xylosidase activity and polynucleotides encoding same
WO2012030845A3 (en) Polypeptides having beta-glucosidase activity, beta-xylosidase activity, or beta-glucosidase and beta-xylosidase activity and polynucleotides encoding same
WO2013163590A3 (en) Gh61 polypeptide variants and polynucleotides encoding same

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 11784356

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 11784356

Country of ref document: EP

Kind code of ref document: A2