CN104602714A - 用于调节bdnf表达的组合物和方法 - Google Patents

用于调节bdnf表达的组合物和方法 Download PDF

Info

Publication number
CN104602714A
CN104602714A CN201380037592.8A CN201380037592A CN104602714A CN 104602714 A CN104602714 A CN 104602714A CN 201380037592 A CN201380037592 A CN 201380037592A CN 104602714 A CN104602714 A CN 104602714A
Authority
CN
China
Prior art keywords
oligonucleotide
nucleotides
nucleotide
stranded oligonucleotide
sequence
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.)
Pending
Application number
CN201380037592.8A
Other languages
English (en)
Chinese (zh)
Inventor
阿瑟·M·克里格
罗梅什·苏布拉马尼安
詹姆斯·麦克斯维根
珍妮·T·李
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.)
General Hospital Corp
Translate Bio Inc
Original Assignee
General Hospital Corp
RaNA Therapeutics Inc
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 General Hospital Corp, RaNA Therapeutics Inc filed Critical General Hospital Corp
Publication of CN104602714A publication Critical patent/CN104602714A/zh
Pending legal-status Critical Current

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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN201380037592.8A 2012-05-16 2013-05-16 用于调节bdnf表达的组合物和方法 Pending CN104602714A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648058P 2012-05-16 2012-05-16
US61/648,058 2012-05-16
PCT/US2013/041385 WO2013173601A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating bdnf expression

Publications (1)

Publication Number Publication Date
CN104602714A true CN104602714A (zh) 2015-05-06

Family

ID=49584294

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201380037592.8A Pending CN104602714A (zh) 2012-05-16 2013-05-16 用于调节bdnf表达的组合物和方法

Country Status (9)

Country Link
US (1) US20150133529A1 (https=)
EP (1) EP2849800A4 (https=)
JP (1) JP2015518711A (https=)
CN (1) CN104602714A (https=)
AU (1) AU2013262702A1 (https=)
BR (1) BR112014028647A2 (https=)
CA (1) CA2873772A1 (https=)
EA (1) EA201492117A1 (https=)
WO (1) WO2013173601A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108315350A (zh) * 2018-03-01 2018-07-24 昆明医科大学 过表达cox5a/低表达bdnf转基因鼠模型及其构建方法与应用

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
JP2016521556A (ja) * 2013-06-07 2016-07-25 ラナ セラピューティクス インコーポレイテッド Ｆｏｘｐ３発現を調節するための組成物及び方法
JP2016531570A (ja) * 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
US11236338B2 (en) * 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
CA2957618A1 (en) * 2014-08-04 2016-02-11 MiRagen Therapeutics, Inc. Inhibitors of myh7b and uses thereof
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
TWI746590B (zh) 2016-06-30 2021-11-21 日商協和麒麟股份有限公司 核酸複合體
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2021262919A2 (en) 2020-06-26 2021-12-30 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
WO2022026648A1 (en) * 2020-07-29 2022-02-03 The General Hospital Corporation Inhibition of incexact1 to treat heart disease
EP4353825A1 (en) * 2022-10-10 2024-04-17 bisy GmbH Modified promoter sequences

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102387817A (zh) * 2009-02-12 2012-03-21 欧科库尔纳有限责任公司 通过抑制针对脑衍生神经营养因子(bdnf)的天然反义转录物来治疗bdnf相关的疾病

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IE921315A1 (en) * 1991-07-03 1993-01-13 Regeneron Pharma Method and assay system for neurothrophin activity
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7715989B2 (en) * 1998-04-03 2010-05-11 Elitech Holding B.V. Systems and methods for predicting oligonucleotide melting temperature (TmS)
US6825338B2 (en) * 2001-03-30 2004-11-30 Isis Pharmaceuticals, Inc. Labeled oligonucleotides, methods for making same, and compounds useful therefor
US20050176025A1 (en) * 2001-05-18 2005-08-11 Sirna Therapeutics, Inc. RNA interference mediated inhibition of B-cell CLL/Lymphoma-2 (BCL-2) gene expression using short interfering nucleic acid (siNA)
AU2003225495B2 (en) * 2002-04-05 2009-01-15 Roche Innovation Center Copenhagen A/S Oligomeric compounds for the modulation HIF-1alpha expression
AU2003291755A1 (en) * 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
WO2008036282A1 (en) * 2006-09-18 2008-03-27 The Regents Of The University Of Californina Upregulating activity or expression of bdnf to mitigate cognitive impairment in asymptomatic huntington's subjects
US20080139472A1 (en) * 2006-10-06 2008-06-12 The Regents Of The University Of California Upregulating bdnf levels to mitigate mental retardation
KR101138048B1 (ko) * 2009-11-06 2012-04-23 성균관대학교산학협력단 Ｂｄｎｆ의 발현을 증가시키는 신규 펩타이드 및 이를 포함하는 알츠하이머병 또는 파킨슨병의 예방 및 치료용 약학 조성물
KR101235256B1 (ko) * 2010-09-13 2013-02-21 서울대학교산학협력단 ｍｉＲＮＡ를 타겟으로 한 신경퇴행성 질환 치료
EP2655621B1 (en) * 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
HK1210211A1 (en) * 2012-03-15 2016-04-15 科纳公司 Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102387817A (zh) * 2009-02-12 2012-03-21 欧科库尔纳有限责任公司 通过抑制针对脑衍生神经营养因子(bdnf)的天然反义转录物来治疗bdnf相关的疾病

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JING ZHAO等: "Genome-wide Identification of Polycomb-Associated RNAs by RIP-seq,", 《MOLECULAR CELL》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108315350A (zh) * 2018-03-01 2018-07-24 昆明医科大学 过表达cox5a/低表达bdnf转基因鼠模型及其构建方法与应用
CN108315350B (zh) * 2018-03-01 2021-08-27 昆明医科大学 过表达cox5a/低表达bdnf转基因鼠模型及其构建方法与应用

Also Published As

Publication number Publication date
US20150133529A1 (en) 2015-05-14
AU2013262702A1 (en) 2015-01-22
EA201492117A1 (ru) 2015-04-30
BR112014028647A2 (pt) 2017-07-25
CA2873772A1 (en) 2013-11-21
WO2013173601A1 (en) 2013-11-21
JP2015518711A (ja) 2015-07-06
EP2849800A1 (en) 2015-03-25
EP2849800A4 (en) 2015-12-09

Similar Documents

Publication Publication Date Title
US11788089B2 (en) Compositions and methods for modulating MECP2 expression
CN104602714A (zh) 用于调节bdnf表达的组合物和方法
US10174315B2 (en) Compositions and methods for modulating hemoglobin gene family expression
US10058623B2 (en) Compositions and methods for modulating UTRN expression
CN104583400A (zh) 用于调节pten表达的组合物和方法
US10174323B2 (en) Compositions and methods for modulating ATP2A2 expression
CN104582737A (zh) 用于调节apoa1和abca1表达的组合物和方法
DK2850186T3 (en) COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
US20160122760A1 (en) Compositions and methods for modulating foxp3 expression
US20150141320A1 (en) Compositions and methods for modulating gene expression

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20150506