WO2015020993A3 - RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA - Google Patents

RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA Download PDF

Info

Publication number
WO2015020993A3
WO2015020993A3 PCT/US2014/049677 US2014049677W WO2015020993A3 WO 2015020993 A3 WO2015020993 A3 WO 2015020993A3 US 2014049677 W US2014049677 W US 2014049677W WO 2015020993 A3 WO2015020993 A3 WO 2015020993A3
Authority
WO
WIPO (PCT)
Prior art keywords
ataxia
methods
friedreich
treatment
rnai compositions
Prior art date
Application number
PCT/US2014/049677
Other languages
French (fr)
Other versions
WO2015020993A2 (en
Inventor
Robert B. Wilson
Yongping Wang
Maria Grazia Cotticelli
Fabio ACQUAVIVA
Shujuan XIA
Original Assignee
The Trustees Of The University Of Pennsylvania
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 Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of WO2015020993A2 publication Critical patent/WO2015020993A2/en
Publication of WO2015020993A3 publication Critical patent/WO2015020993A3/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
    • C12N15/11—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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
    • C12N2330/31—Libraries, arrays

Landscapes

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

Abstract

The invention provides ribonucleic acid (RNA) molecules, such as siRNA and shRNA molecules, that compensate for a frataxin deficiency or mutation, expression vectors encoding the same, and methods of using the same (e.g., to treat Friedreich's ataxia).
PCT/US2014/049677 2013-08-05 2014-08-05 RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA WO2015020993A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361862254P 2013-08-05 2013-08-05
US61/862,254 2013-08-05

Publications (2)

Publication Number Publication Date
WO2015020993A2 WO2015020993A2 (en) 2015-02-12
WO2015020993A3 true WO2015020993A3 (en) 2015-11-05

Family

ID=52462029

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/049677 WO2015020993A2 (en) 2013-08-05 2014-08-05 RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA

Country Status (1)

Country Link
WO (1) WO2015020993A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9163231B2 (en) 2006-03-07 2015-10-20 The Trustees Of The University Of Pennsylvania Random RNAi libraries, methods of generating same, and screening methods utilizing same
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
EP3256591A4 (en) 2015-02-13 2018-08-08 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070116691A1 (en) * 2005-10-18 2007-05-24 National Jewish Medical And Research Center Conditionally immortalized long-term stem cells and methods of making and using such cells
US20120308645A1 (en) * 2002-11-01 2012-12-06 The Trustees Of The University Of Pennsylvania Compositions and methods for sirna inhibition of hif-1 alpha
US20130096183A1 (en) * 2010-06-23 2013-04-18 Curna, Inc. Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120308645A1 (en) * 2002-11-01 2012-12-06 The Trustees Of The University Of Pennsylvania Compositions and methods for sirna inhibition of hif-1 alpha
US20070116691A1 (en) * 2005-10-18 2007-05-24 National Jewish Medical And Research Center Conditionally immortalized long-term stem cells and methods of making and using such cells
US20130096183A1 (en) * 2010-06-23 2013-04-18 Curna, Inc. Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"CW908201, RPCI42_148G4.TJ RPCI-42 Bos taurus genomic clone RPCI42_148G4, genomic survey sequence", 8 December 2004 (2004-12-08), Retrieved from the Internet <URL:http://www.ncbi.nlm.nih.gov/nucgss/CW908201> [retrieved on 20150225] *

Also Published As

Publication number Publication date
WO2015020993A2 (en) 2015-02-12

Similar Documents

Publication Publication Date Title
ZA202110854B (en) Tmprss6 irna compositions and methods of use thereof
MX2018006989A (en) Methods and compositions for treating a serpinc1-associated disorder.
PH12017500669A1 (en) Compositions and methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
MX2021009118A (en) Compositions and methods for inhibiting expression of the alas1 gene.
MX2019008380A (en) Complement component c5 irna compositions and methods of use thereof.
MX2015008697A (en) Age-related macular degeneration treatment.
PH12014501106B1 (en) Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
MX2018013216A (en) Compositions and methods for inhibiting expression of tmprss6 gene.
MX2019006413A (en) Serpinc1 irna compositions and methods of use thereof.
MX2021000656A (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF.
MX2021010716A (en) Compositions and methods for inhibiting expression of the lect2 gene.
MX2022001017A (en) Compositions and methods for inhibiting expression of the alas1 gene.
WO2014093746A3 (en) Methods and compositions for the specific inhibition of ckap5 by double-stranded rna
WO2015020993A3 (en) RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH&#39;S ATAXIA
MX2019007110A (en) Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof.
MX368084B (en) Sirna and their use in methods and compositions for the treatment and/or prevention of eye conditions.
TW202423457A (en) Methods and compositions for treating a serpinc1-associated disorder
TN2014000211A1 (en) Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
MY185464A (en) Complement component c5 irna compositions and methods of use thereof
EA037110B9 (en) PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

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: 14835398

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: 14835398

Country of ref document: EP

Kind code of ref document: A2