CA2690730C - Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease - Google Patents

Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease Download PDF

Info

Publication number
CA2690730C
CA2690730C CA2690730A CA2690730A CA2690730C CA 2690730 C CA2690730 C CA 2690730C CA 2690730 A CA2690730 A CA 2690730A CA 2690730 A CA2690730 A CA 2690730A CA 2690730 C CA2690730 C CA 2690730C
Authority
CA
Canada
Prior art keywords
acid molecule
short interfering
ribonucleic acid
double
interfering ribonucleic
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Fee Related
Application number
CA2690730A
Other languages
English (en)
French (fr)
Other versions
CA2690730A1 (en
Inventor
Valerie Perrin
Nicole Deglon
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Commissariat a lEnergie Atomique et aux Energies Alternatives CEA
Original Assignee
Commissariat a lEnergie Atomique CEA
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 Commissariat a lEnergie Atomique CEA filed Critical Commissariat a lEnergie Atomique CEA
Publication of CA2690730A1 publication Critical patent/CA2690730A1/en
Application granted granted Critical
Publication of CA2690730C publication Critical patent/CA2690730C/en
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CA2690730A 2007-06-18 2008-06-18 Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease Expired - Fee Related CA2690730C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP07290751A EP2014769B1 (en) 2007-06-18 2007-06-18 Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
EP07290751.2 2007-06-18
PCT/IB2008/002603 WO2009007855A2 (en) 2007-06-18 2008-06-18 Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease

Publications (2)

Publication Number Publication Date
CA2690730A1 CA2690730A1 (en) 2009-01-15
CA2690730C true CA2690730C (en) 2018-07-31

Family

ID=38476952

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2690730A Expired - Fee Related CA2690730C (en) 2007-06-18 2008-06-18 Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease

Country Status (7)

Country Link
US (1) US8217018B2 (enExample)
EP (1) EP2014769B1 (enExample)
JP (1) JP5401451B2 (enExample)
AT (1) ATE462787T1 (enExample)
CA (1) CA2690730C (enExample)
DE (1) DE602007005629D1 (enExample)
WO (1) WO2009007855A2 (enExample)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2297341A4 (en) * 2008-05-09 2013-01-09 Univ British Columbia METHOD AND COMPOSITIONS FOR TREATING MORBUS HUNTINGTON
US8679750B2 (en) 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011097643A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2591356A1 (en) * 2010-07-09 2013-05-15 Ecole Polytechnique Fédérale de Lausanne (EPFL) Method for in-vitro monitoring of neuronal disorders and use thereof
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
JP6128529B2 (ja) 2011-07-19 2017-05-17 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. 官能化核酸の合成のための方法
ES2651514T3 (es) 2011-08-11 2018-01-26 Ionis Pharmaceuticals, Inc. Compuestos oligoméricos gapados que comprenden deoxirribonucleósidos modificados en 5 ' -en la brecha y usos de ellos
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
MX356830B (es) 2012-07-13 2018-06-15 Shin Nippon Biomedical Laboratories Ltd Adyuvante de acido nucleico quiral.
US9982257B2 (en) 2012-07-13 2018-05-29 Wave Life Sciences Ltd. Chiral control
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
KR20150070278A (ko) * 2012-10-15 2015-06-24 아이시스 파마수티컬즈 인코포레이티드 C9orf72 발현 조절용 조성물
JP2016516804A (ja) 2013-04-17 2016-06-09 ファイザー・インク 心血管疾患を治療するためのn−ピペリジン−3−イルベンズアミド誘導体
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
US10369201B2 (en) * 2013-11-11 2019-08-06 Sangamo Therapeutics, Inc. Methods and compositions for treating Huntington's disease
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
WO2015108046A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
DK3094728T3 (da) 2014-01-16 2022-05-16 Wave Life Sciences Ltd Kiralt design
CA2947035A1 (en) * 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
SMT202000165T1 (it) 2015-04-16 2020-05-08 Ionis Pharmaceuticals Inc Composizioni per modulare l'espressione di c9orf72
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés

Also Published As

Publication number Publication date
CA2690730A1 (en) 2009-01-15
US8217018B2 (en) 2012-07-10
JP5401451B2 (ja) 2014-01-29
EP2014769B1 (en) 2010-03-31
ATE462787T1 (de) 2010-04-15
EP2014769A1 (en) 2009-01-14
WO2009007855A3 (en) 2009-04-02
JP2010530239A (ja) 2010-09-09
US20100299768A1 (en) 2010-11-25
DE602007005629D1 (de) 2010-05-12
WO2009007855A2 (en) 2009-01-15

Similar Documents

Publication Publication Date Title
CA2690730C (en) Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease
AU2015264263B2 (en) Huntington's disease therapeutic compounds
US8779116B2 (en) SiRNA-mediated gene silencing
US8524879B2 (en) RNA interference suppresion of neurodegenerative diseases and methods of use thereof
JP3642573B2 (ja) siRNA発現システムおよびこれを用いた機能遺伝子ノックダウン細胞等の生産方法
US20040002077A1 (en) siRNA expression system and method for producing functional gene knock-down cell using the system
ZA200501020B (en) Allele-specific siRNA-mediated gene silencing
US20100086526A1 (en) Nucleic acid constructs and methods for specific silencing of h19
IL194114A (en) Use of RNA compound interferes with the preparation of drugs for the treatment of central nervous system disorder, medicinal preparations for intraperitoneal administration containing one or more SINAs directed to the gene expressed in the central nervous system.
JP2010512794A (ja) 核酸の発現のための組成物及び方法
Cazzin Development of viral vector-mediated RNA interference targeting several P2X receptors in the rat central nervous system
HK1236224B (en) Huntington's disease therapeutic compounds
HK1236224A1 (en) Huntington's disease therapeutic compounds
EP1581635A2 (en) Sirna-mediated gene silencing

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20130529

MKLA Lapsed

Effective date: 20220301

MKLA Lapsed

Effective date: 20200831