CA2726866A1 - Procedes et compositions pour le traitement de la maladie de huntington - Google Patents

Procedes et compositions pour le traitement de la maladie de huntington Download PDF

Info

Publication number
CA2726866A1
CA2726866A1 CA2726866A CA2726866A CA2726866A1 CA 2726866 A1 CA2726866 A1 CA 2726866A1 CA 2726866 A CA2726866 A CA 2726866A CA 2726866 A CA2726866 A CA 2726866A CA 2726866 A1 CA2726866 A1 CA 2726866A1
Authority
CA
Canada
Prior art keywords
nucleic acid
cag
allele
polymorphism
chromosomes
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.)
Abandoned
Application number
CA2726866A
Other languages
English (en)
Inventor
Michael Hayden
Jeffrey Carroll
Simon Warby
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.)
University of British Columbia
Original Assignee
University of British Columbia
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 University of British Columbia filed Critical University of British Columbia
Publication of CA2726866A1 publication Critical patent/CA2726866A1/fr
Abandoned legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
CA2726866A 2008-05-09 2009-05-08 Procedes et compositions pour le traitement de la maladie de huntington Abandoned CA2726866A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US71465208P 2008-05-09 2008-05-09
US61/0714,652 2008-05-09
PCT/CA2009/000645 WO2009135322A1 (fr) 2008-05-09 2009-05-08 Procédés et compositions pour le traitement de la maladie de huntington

Publications (1)

Publication Number Publication Date
CA2726866A1 true CA2726866A1 (fr) 2009-11-12

Family

ID=41264389

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2726866A Abandoned CA2726866A1 (fr) 2008-05-09 2009-05-08 Procedes et compositions pour le traitement de la maladie de huntington

Country Status (4)

Country Link
EP (1) EP2297341A4 (fr)
AU (1) AU2009244013B2 (fr)
CA (1) CA2726866A1 (fr)
WO (1) WO2009135322A1 (fr)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2203173B1 (fr) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Moyens et procédé de compensation des troubles musculaires
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
US8957040B2 (en) * 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
JP6018506B2 (ja) * 2010-02-08 2016-11-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 対立遺伝子多様体の選択的低減
WO2012039448A1 (fr) 2010-09-24 2012-03-29 株式会社キラルジェン Groupe auxiliaire asymétrique
EP2734208B1 (fr) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Procédés pour la synthèse d'acides nucléiques fonctionnalisés
US10202599B2 (en) 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
EP2872485B1 (fr) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Groupe auxiliaire asymétrique
WO2014010718A1 (fr) 2012-07-13 2014-01-16 株式会社新日本科学 Adjuvant d'acide nucléique chiral
US20150275208A1 (en) 2012-10-12 2015-10-01 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2014121287A2 (fr) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Composés antisens sélectifs et leurs utilisations
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 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
WO2015108047A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
AU2015207773B2 (en) 2014-01-16 2021-06-17 Wave Life Sciences Ltd. Chiral design
WO2015164693A1 (fr) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. COMPOSÉS OLIGOMÈRES COMPRENANT UN ACIDE NUCLÉIQUE À CONFORMATION CONTRAINTE α-β
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2021113769A1 (fr) * 2019-12-07 2021-06-10 Scribe Therapeutics Inc. Compositions et méthodes pour le ciblage de htt

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020187931A1 (en) * 2000-04-13 2002-12-12 Michael Hayden Modulating cell survival by modulating huntingtin function
WO2008005562A2 (fr) * 2006-07-07 2008-01-10 University Of Massachusetts Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington
EP2062980B1 (fr) * 2005-06-28 2011-08-31 Medtronic, Inc. Méthodes et séquences pour supprimer préférentiellement l'expression du gène huntingtin muté.
US9273356B2 (en) * 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
EP2014769B1 (fr) * 2007-06-18 2010-03-31 Commissariat à l'Energie Atomique Amortissement du bruit à base de sirna réversible de gène huntingtin de type sauvage endogènes et mutés et son application pour le traitement de la maladie de Hungtington

Also Published As

Publication number Publication date
EP2297341A4 (fr) 2013-01-09
AU2009244013B2 (en) 2015-06-25
AU2009244013A1 (en) 2009-11-12
EP2297341A1 (fr) 2011-03-23
WO2009135322A1 (fr) 2009-11-12

Similar Documents

Publication Publication Date Title
US9157120B2 (en) Methods and compositions for the treatment of huntington's disease
AU2009244013B2 (en) Methods and compositions for the treatment of Huntington's disease
Karban et al. Functional annotation of a novel NFKB1 promoter polymorphism that increases risk for ulcerative colitis
US20210388444A1 (en) Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases
Feng et al. Evidence for X-chromosomal schizophrenia associated with microRNA alterations
Xu et al. A heroin addiction severity-associated intronic single nucleotide polymorphism modulates alternative pre-mRNA splicing of the μ opioid receptor gene OPRM1 via hnRNPH interactions
US10266896B2 (en) Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis and treatment of type 1 diabetes
Wei et al. MicroRNA-146a and Ets-1 gene polymorphisms are associated with pediatric uveitis
Li et al. A SNP in pri-miR-10a is associated with recurrent spontaneous abortion in a Han-Chinese population
Argmann et al. Germline deletion of Krüppel-like factor 14 does not increase risk of diet induced metabolic syndrome in male C57BL/6 mice
JP6095889B2 (ja) 染色体２１ｑ、６ｑ、および１５ｑの遺伝子変異およびこれらを使用して１型糖尿病を診断および治療する方法
US9926600B2 (en) Genetic alterations associated with type I diabetes and methods for use thereof for diagnosis and treatment
Murmann et al. The length of uninterrupted CAG repeats in stem regions of repeat disease associated hairpins determines the amount of short CAG oligonucleotides that are toxic to cells through RNA interference
US10731160B2 (en) Allele-specific therapy for Huntington disease haplotypes
US11674140B2 (en) Compositions and methods for treating facioscapulohumeral dystrophy
US20230383351A1 (en) Rare Variants In Hematopoietic Stem Cells (HSC) And Hematopoietic Progenitor Cells (HPC) Associated With Somatic Alterations Of The Blood
Morton The length of uninterrupted CAG repeats in stem regions of repeat disease associated hairpins determines the amount of short CAG oligonucleotides that are toxic to cells through RNA interferenc
WO2023141577A2 (fr) Méthodes d'amélioration de la santé avec des inhibiteurs d'apolipoprotéine e (apoe)
WO2022216832A1 (fr) Composition destinée à être utilisée dans le traitement du cmh-1-opathies
CN116139276A (zh) Rnf130及其下调剂在制备自身免疫性疾病药物中的应用
WO2010022235A2 (fr) Une étude d'association à l'échelle du génome sur l'autisme révèle un nouveau locus à risque commun sur 5p14.1

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20140414

FZDE Dead

Effective date: 20171019