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WO2007135426A3 - Treatment of neurodegeneratives diseases - Google Patents

Treatment of neurodegeneratives diseases Download PDF

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Publication number
WO2007135426A3
WO2007135426A3 PCT/GB2007/001900 GB2007001900W WO2007135426A3 WO 2007135426 A3 WO2007135426 A3 WO 2007135426A3 GB 2007001900 W GB2007001900 W GB 2007001900W WO 2007135426 A3 WO2007135426 A3 WO 2007135426A3
Authority
WO
WIPO (PCT)
Prior art keywords
treatment
synuclein
neurodegeneratives
diseases
neurodegeneratives diseases
Prior art date
Application number
PCT/GB2007/001900
Other languages
French (fr)
Other versions
WO2007135426A2 (en
Inventor
Richard Wade-Martins
Tim Fountaine
Original Assignee
Isis Innovation
Richard Wade-Martins
Tim Fountaine
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 Isis Innovation, Richard Wade-Martins, Tim Fountaine filed Critical Isis Innovation
Publication of WO2007135426A2 publication Critical patent/WO2007135426A2/en
Publication of WO2007135426A3 publication Critical patent/WO2007135426A3/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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.

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention is directed to the use of an agent that down regulates α-synuclein to down regulate α-synuclein in a subject with normal physiological levels of α-synuclein expression. In particular, for the treatment of neurodegenerative disease, such as sporadic Parkinson's disease.
PCT/GB2007/001900 2006-05-23 2007-05-23 Treatment of neurodegeneratives diseases WO2007135426A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0610183.6A GB0610183D0 (en) 2006-05-23 2006-05-23 Treatment of neurodegenerative diseases
GB0610183.6 2006-05-23

Publications (2)

Publication Number Publication Date
WO2007135426A2 WO2007135426A2 (en) 2007-11-29
WO2007135426A3 true WO2007135426A3 (en) 2008-01-31

Family

ID=36660655

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2007/001900 WO2007135426A2 (en) 2006-05-23 2007-05-23 Treatment of neurodegeneratives diseases

Country Status (2)

Country Link
GB (1) GB0610183D0 (en)
WO (1) WO2007135426A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129791A1 (en) 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
EP2380595A1 (en) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
US9663783B2 (en) 2010-11-17 2017-05-30 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP2721145A4 (en) * 2011-06-16 2014-12-10 Parkinson S Inst High through-put screening in skin fibroblasts with an alpha-synuclein triplication
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
EP3377629B1 (en) 2015-11-18 2023-07-05 Rosalind Franklin University of Medicine and Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
WO2018080573A1 (en) * 2016-10-28 2018-05-03 Massachusetts Institute Of Technology Crispr/cas global regulator screening platform
TWI809004B (en) 2017-11-09 2023-07-21 美商Ionis製藥公司 Compounds and methods for reducing snca expression
JP7455746B2 (en) 2018-01-12 2024-03-26 ブリストル－マイヤーズ スクイブ カンパニー Antisense oligonucleotides targeting alpha-synuclein and their uses
BR112020012921A2 (en) 2018-01-12 2020-12-08 Roche Innovation Center Copenhagen A/S ALPHA-SINUCLEIN ANTISENSE OLIGONUCLEOTIDES AND USES THEREOF

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
WO2006039253A2 (en) * 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
WO2006039253A2 (en) * 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
FOUNTAINE TIMOTHY M ET AL: "Delivering RNA interference to the mammalian brain", CURRENT GENE THERAPY, XX, XX, vol. 5, no. 4, August 2005 (2005-08-01), pages 399 - 410, XP009092374, ISSN: 1566-5232 *
FOUNTAINE TIMOTHY M ET AL: "RNA interference-mediated knockdown of alpha-synuclein protects human dopaminergic neuroblastoma cells from MPP+ toxicity and reduces dopamine transport", JOURNAL OF NEUROSCIENCE RESEARCH, vol. 85, no. 2, February 2007 (2007-02-01), pages 351 - 363, XP002459531, ISSN: 0360-4012 *
HAYASHITA-KINOH ET AL: "Down-regulation of alpha-synuclein expression can rescue dopaminergic cells from cell death in the substantia nigra of Parkinson's disease rat model", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, ACADEMIC PRESS INC. ORLANDO, FL, US, vol. 341, no. 4, 24 March 2006 (2006-03-24), pages 1088 - 1095, XP005282999, ISSN: 0006-291X *
SAPRU ET AL: "Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi", EXPERIMENTAL NEUROLOGY, ACADEMIC PRESS, NEW YORK, NY, US, vol. 198, no. 2, April 2006 (2006-04-01), pages 382 - 390, XP005358981, ISSN: 0014-4886 *

Also Published As

Publication number Publication date
WO2007135426A2 (en) 2007-11-29
GB0610183D0 (en) 2006-06-28

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