WO2015179656A3 - Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr) - Google Patents

Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr) Download PDF

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Publication number
WO2015179656A3
WO2015179656A3 PCT/US2015/031995 US2015031995W WO2015179656A3 WO 2015179656 A3 WO2015179656 A3 WO 2015179656A3 US 2015031995 W US2015031995 W US 2015031995W WO 2015179656 A3 WO2015179656 A3 WO 2015179656A3
Authority
WO
WIPO (PCT)
Prior art keywords
cftr
cystic fibrosis
methods
cell
conductance regulator
Prior art date
Application number
PCT/US2015/031995
Other languages
French (fr)
Other versions
WO2015179656A2 (en
Inventor
Kevin Morris
Sheena SAAYMAN
Original Assignee
The Scripps Research Institute
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 The Scripps Research Institute filed Critical The Scripps Research Institute
Publication of WO2015179656A2 publication Critical patent/WO2015179656A2/en
Publication of WO2015179656A3 publication Critical patent/WO2015179656A3/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

This invention provides compositions and methods for enhancing CFTR expression in a cell. The invention also provides methods for treating cystic fibrosis. In one aspect, the invention provides methods for enhancing expression or cellular level of cystic fibrosis transmembrane conductance regulator (CFTR) in a cell. The methods entail contacting the cell with an agent that specifically disrupts or downregulates CFTR associated antisense long non-coding RNA (IncRNA) BG213071 and/or A1805947. Some of the methods are directed enhancing CFTR expression in an epithelial cell.
PCT/US2015/031995 2014-05-23 2015-05-21 Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr) WO2015179656A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462002462P 2014-05-23 2014-05-23
US62/002,462 2014-05-23

Publications (2)

Publication Number Publication Date
WO2015179656A2 WO2015179656A2 (en) 2015-11-26
WO2015179656A3 true WO2015179656A3 (en) 2016-01-14

Family

ID=54554972

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2015/031995 WO2015179656A2 (en) 2014-05-23 2015-05-21 Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr)

Country Status (1)

Country Link
WO (1) WO2015179656A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017027814A1 (en) * 2015-08-13 2017-02-16 Arcturus Therapeutics, Inc. Una oligomeric agents for stimulating cystic fibrosis transmembrane conductance regulator and uses thereof
WO2022008557A2 (en) * 2020-07-08 2022-01-13 UCB Biopharma SRL Modulation of cftr expression

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050272080A1 (en) * 2004-05-03 2005-12-08 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
US20130145485A1 (en) * 2011-07-25 2013-06-06 The University Of Texas Health Science Center At Houston Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
WO2013173635A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050272080A1 (en) * 2004-05-03 2005-12-08 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
US20130145485A1 (en) * 2011-07-25 2013-06-06 The University Of Texas Health Science Center At Houston Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
WO2013173635A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
DATABASE GenBank [o] 7 July 1999 (1999-07-07), "te52h07.x1 Soares_NFL_T_GBC_S1 Homo sapiens cDNA clone, mRNA sequence", Database accession no. AI805947.1 *
ENTEZARI ET AL.: "Inhibition of high-mobility group box 1 protein (HMGB1) enhances bacterial clearance and protects against Pseudomonas Aeruginosa pneumonia in cystic fibrosis", MOL MED., vol. 18, 9 May 2012 (2012-05-09), pages 477 - 485 *
ROBERTS ET AL.: "Perspectives on the mechanism of transcriptional regulation by long non-coding RNAs", EPIGENETICS, vol. 9, no. Iss. 1, 1 January 2014 (2014-01-01), pages 13 - 20 *
SAAYMAN ET AL.: "Abstract No. 10. Cystic Fibrosis Transmembrane Receptor Expression is Regulated by Long Antisense Non-Coding RNAs", MOLECULAR THERAPY, vol. 22, 1 May 2014 (2014-05-01), pages S4 *
SAAYMAN ET AL.: "An HIV-encoded antisense long noncoding RNA epigenetically regulates viral transcription", MOL THER., vol. 22, 28 February 2014 (2014-02-28), pages 1164 - 1175 *

Also Published As

Publication number Publication date
WO2015179656A2 (en) 2015-11-26

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