PL3074050T3 - Kompozycje i sposoby modulowania pobudliwości neuronalnej i zachowania motorycznego - Google Patents

Kompozycje i sposoby modulowania pobudliwości neuronalnej i zachowania motorycznego

Info

Publication number
PL3074050T3
PL3074050T3 PL14857152T PL14857152T PL3074050T3 PL 3074050 T3 PL3074050 T3 PL 3074050T3 PL 14857152 T PL14857152 T PL 14857152T PL 14857152 T PL14857152 T PL 14857152T PL 3074050 T3 PL3074050 T3 PL 3074050T3
Authority
PL
Poland
Prior art keywords
compositions
methods
motor behavior
neuronal excitability
modulating neuronal
Prior art date
Application number
PL14857152T
Other languages
English (en)
Inventor
Anne Schaefer
Paul Greengard
Original Assignee
Icahn School Of Medicine At Mount Sinai
The Rockefeller University
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 Icahn School Of Medicine At Mount Sinai, The Rockefeller University filed Critical Icahn School Of Medicine At Mount Sinai
Publication of PL3074050T3 publication Critical patent/PL3074050T3/pl

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    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knockout animals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058—Neurological cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/203—Animal model comprising inducible/conditional expression system, e.g. hormones, tet
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • 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
    • 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.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
PL14857152T 2013-10-28 2014-10-28 Kompozycje i sposoby modulowania pobudliwości neuronalnej i zachowania motorycznego PL3074050T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361896463P 2013-10-28 2013-10-28
US201361898952P 2013-11-01 2013-11-01
EP14857152.4A EP3074050B1 (en) 2013-10-28 2014-10-28 Compositions and methods for modulating neuronal excitability and motor behavior
PCT/US2014/062664 WO2015066034A1 (en) 2013-10-28 2014-10-28 Compositions and methods for modulating neuronal excitability and motor behavior

Publications (1)

Publication Number Publication Date
PL3074050T3 true PL3074050T3 (pl) 2021-04-06

Family

ID=53005028

Family Applications (1)

Application Number Title Priority Date Filing Date
PL14857152T PL3074050T3 (pl) 2013-10-28 2014-10-28 Kompozycje i sposoby modulowania pobudliwości neuronalnej i zachowania motorycznego

Country Status (8)

Country Link
US (3) US10087443B2 (pl)
EP (2) EP3825402A1 (pl)
JP (2) JP6700180B2 (pl)
AU (2) AU2014342535B2 (pl)
CA (1) CA2929098A1 (pl)
ES (1) ES2836133T3 (pl)
PL (1) PL3074050T3 (pl)
WO (1) WO2015066034A1 (pl)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101814868B1 (ko) * 2015-06-18 2018-01-04 재단법인대구경북과학기술원 마이크로 rna와 nmda 수용체의 상관관계를 이용한 해마의 기능감소 판단 방법, 기능감소 억제 방법 및 기능감소 억제제 스크리닝 방법
JP6923509B2 (ja) * 2015-07-28 2021-08-18 オトノミ—，インク． 切断型のｔｒｋｂおよびｔｒｋｃのアンタゴニストを使用する処置
US11230720B2 (en) 2015-10-14 2022-01-25 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
US11236337B2 (en) 2016-11-01 2022-02-01 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
CA3042401A1 (en) * 2016-11-01 2018-05-11 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
WO2019227255A1 (zh) * 2018-05-26 2019-12-05 深圳市博奥康生物科技有限公司 miRNA-128过表达载体的构建及其应用

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US5830857A (en) * 1995-07-14 1998-11-03 Amgen Inc. Method of treating epilepsy
IL128941A (en) * 1996-09-13 2007-05-15 Advanced Medicine Res Inst An eye preparation that contains a neurotrophic factor for the treatment of a functional disease of the optic nerve
WO2006070292A2 (en) * 2004-10-12 2006-07-06 Queen's University At Kingston Pyrimidine compounds as anti-ictogenic and/or anti-epileptogenic agents
CA2850323A1 (en) * 2004-11-12 2006-12-28 Asuragen, Inc. Methods and compositions involving mirna and mirna inhibitor molecules
EP2623095A1 (en) * 2004-11-16 2013-08-07 Elan Pharma International Limited Injectable nanoparticulate olanzapine formulations
JP2011528227A (ja) * 2008-07-16 2011-11-17 ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ 再有髄化におけるｇｍ９８（ｍｒｆ）の調節
CA2808372C (en) * 2010-08-16 2021-11-16 Brainstem Biotec Ltd. Methods of generating oligodendrocytes and cell populations comprising same
EP2663323B1 (en) 2011-01-14 2017-08-16 The General Hospital Corporation Methods targeting mir-128 for regulating cholesterol/lipid metabolism
WO2013045652A1 (en) * 2011-09-28 2013-04-04 Royal College Of Surgeons In Ireland Inhibition of microrna-134 for the treatment of seizure-related disorders and neurologic injuries
JP5850702B2 (ja) * 2011-10-25 2016-02-03 国立大学法人岐阜大学 間葉系細胞の分化調節剤およびこれを用いた医薬、並びに間葉系細胞への分化調節作用を有する物質のスクリーニング方法
GB201223244D0 (en) 2012-12-21 2013-02-06 Ucb Pharma Gmbh Micrornas as therapeutics and biomarkers for epilepsy
US9434945B2 (en) 2013-08-02 2016-09-06 University Of Maryland, Baltimore Use of miR-23a-3p and/or miR-27a-3p mimics as therapeutic agents for inhibition of neuronal apoptosis following brain injury
CN103656685B (zh) 2014-01-10 2016-01-13 厦门大学 microRNA-219在制备抗癫痫药物中的应用
WO2015175965A1 (en) 2014-05-15 2015-11-19 The Research Foundation For Suny Compositions targeting the interaction domain between p27kip1 and brk and methods of use thereof

Also Published As

Publication number Publication date
JP2016540740A (ja) 2016-12-28
AU2020204419B2 (en) 2023-11-02
EP3074050A4 (en) 2017-11-29
WO2015066034A1 (en) 2015-05-07
JP7014852B2 (ja) 2022-02-01
CA2929098A1 (en) 2015-05-07
US20190144858A1 (en) 2019-05-16
US10870853B2 (en) 2020-12-22
AU2014342535B2 (en) 2020-04-02
ES2836133T3 (es) 2021-06-24
AU2020204419A1 (en) 2020-07-23
EP3825402A1 (en) 2021-05-26
AU2014342535A1 (en) 2016-06-09
US20210317451A1 (en) 2021-10-14
ES2836133T8 (es) 2022-09-09
JP2020158506A (ja) 2020-10-01
EP3074050B1 (en) 2020-09-09
EP3074050A1 (en) 2016-10-05
US10087443B2 (en) 2018-10-02
US20160244757A1 (en) 2016-08-25
JP6700180B2 (ja) 2020-05-27

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