WO2007135426A3 - Treatment of neurodegeneratives diseases - Google Patents

Treatment of neurodegeneratives diseases Download PDF

Info

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.

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
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University 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
CA2817960C (en) * 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US20150037257A1 (en) * 2011-06-16 2015-02-05 Sally Mak Compositions and methods for high-throughput screening in skin fibroblasts with an alpha-synuclein triplication
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
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
US20180119141A1 (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
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
EP3737759A1 (en) 2018-01-12 2020-11-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein 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
GB0610183D0 (en) 2006-06-28
WO2007135426A2 (en) 2007-11-29

Similar Documents

Publication Publication Date Title
WO2007135426A3 (en) Treatment of neurodegeneratives diseases
GB2443287B (en) Methods, compositions and formulations for the treatment of thyroid eye disease
WO2008103472A3 (en) Prevention and treatment of synucleinopathic and amyloidogenic disease
WO2008054200A3 (en) Use of nutritional compositions for preventing disorders
WO2007022506A3 (en) Methods and compositions for treating neurological disease
HK1166983A1 (en) Solid forms comprising compounds for the treatment of diseases or disorders, compositions thereof, and use therewith
WO2008029276A3 (en) Compositions and methods for the treatment of ophthalmic disease
WO2008088537A3 (en) Stent with a coating for delivering a therapeutic agent
MX2009006342A (en) Nutritional supplement composition for treatment of ocular diseases.
EP2091566A4 (en) Combination treatments for alzheimer's disease and similar diseases
PT2056863T (en) Treatment of ocular diseases
HK1110221A1 (en) Use of prodrugs for ocular intravitreous administration
WO2007135026A3 (en) Substituted pteridines as therapeutic agents
WO2007075923A3 (en) Treatment of synucleinopathies
PT2056804E (en) Use of 2,5-dihydroxybenzene derivatives for the treatment of ocular diseases
WO2010064248A3 (en) Methods of diagnosing and treating motor neuron diseases
EP2048141A4 (en) Optical pure dihydropyrimidine compounds, their uses in the manufacture of a medicamnent for the treatment or the prevention of virosis diseases
HK1120830A1 (en) Modulation of 11beta-hydroxysteroid dehydrogenase 1 expression for the treatment of ocular diseases
GB0523961D0 (en) The treatment of ophthalmic diseases
WO2007133983A3 (en) 2-aminobenzimidazoles for treating neurodegenerative diseases
WO2009062750A3 (en) Active soluble post-translationally modified neuregulin isoforms
EP2005194A4 (en) Hemoglobin-based methods for prophylaxis, diagnosis and/or treatment of retinal disorders
WO2007147868A3 (en) Prevention of muscle atrophy
WO2008051421A3 (en) Peptide-cytotoxic conjugates
WO2009074498A8 (en) Medical use of 3- (2, 2, 2-trimethylhydrazinixm) propionate orotate

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: 07732921

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: 07732921

Country of ref document: EP

Kind code of ref document: A2