JP2015523410A5 - - Google Patents

Download PDF

Info

Publication number
JP2015523410A5
JP2015523410A5 JP2015524902A JP2015524902A JP2015523410A5 JP 2015523410 A5 JP2015523410 A5 JP 2015523410A5 JP 2015524902 A JP2015524902 A JP 2015524902A JP 2015524902 A JP2015524902 A JP 2015524902A JP 2015523410 A5 JP2015523410 A5 JP 2015523410A5
Authority
JP
Japan
Prior art keywords
als
agent
mnd
processing
expression
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.)
Granted
Application number
JP2015524902A
Other languages
English (en)
Japanese (ja)
Other versions
JP6340366B2 (ja
JP2015523410A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/IL2013/050655 external-priority patent/WO2014020608A1/en
Publication of JP2015523410A publication Critical patent/JP2015523410A/ja
Publication of JP2015523410A5 publication Critical patent/JP2015523410A5/ja
Application granted granted Critical
Publication of JP6340366B2 publication Critical patent/JP6340366B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2015524902A 2012-07-31 2013-07-31 運動ニューロン疾患を診断および処置する方法 Active JP6340366B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261677494P 2012-07-31 2012-07-31
US61/677,494 2012-07-31
PCT/IL2013/050655 WO2014020608A1 (en) 2012-07-31 2013-07-31 Methods of diagnosing and treating motor neuron diseases

Publications (3)

Publication Number Publication Date
JP2015523410A JP2015523410A (ja) 2015-08-13
JP2015523410A5 true JP2015523410A5 (https=) 2016-06-23
JP6340366B2 JP6340366B2 (ja) 2018-06-06

Family

ID=49085064

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015524902A Active JP6340366B2 (ja) 2012-07-31 2013-07-31 運動ニューロン疾患を診断および処置する方法

Country Status (6)

Country Link
US (1) US10159670B2 (https=)
EP (1) EP2879678B1 (https=)
JP (1) JP6340366B2 (https=)
CN (1) CN104684558A (https=)
HK (1) HK1205453A1 (https=)
WO (1) WO2014020608A1 (https=)

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP6340366B2 (ja) 2012-07-31 2018-06-06 イェダ リサーチ アンド デベロップメント カンパニー リミテッド 運動ニューロン疾患を診断および処置する方法
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3115457B1 (en) * 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016030899A1 (en) * 2014-08-28 2016-03-03 Yeda Research And Development Co. Ltd. Methods of treating amyotrophic lateral scleroses
WO2016138287A1 (en) 2015-02-25 2016-09-01 Washington University METHODS TO DETECT MOTOR NEURON DISEASE COMPRISING MICRO-RNAs
US20180064748A1 (en) 2015-03-27 2018-03-08 Yeda Research And Development Co. Ltd. Methods of treating motor neuron diseases
JP6666002B2 (ja) * 2015-10-07 2020-03-13 国立大学法人京都大学 Tdp−43プロテノパシーの予防又は治療用組成物
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN105663129B (zh) * 2015-12-29 2020-05-15 山东大学 用于治疗肌萎缩侧索硬化和额颞叶痴呆的化合物与应用
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN106620721B (zh) * 2016-09-30 2020-05-26 武汉大风生物科技有限公司 单核细胞趋化蛋白-1诱导蛋白1(Mcpip1)在肝缺血再灌注损伤中的应用
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CN106377761A (zh) * 2016-10-27 2017-02-08 武汉大学 单核细胞趋化蛋白‑1诱导蛋白1在治疗脂肪肝和ⅱ型糖尿病中的功能和应用
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
JP7270255B2 (ja) * 2017-06-20 2023-05-10 ニューロセンス セラピューティックス リミテッド 抗炎症剤及びダイサー活性化剤を含む神経疾患治療用組成物
US12527768B2 (en) 2017-06-20 2026-01-20 Neurosense Therapeutics Ltd. Compositions comprising an anti-inflammatory drug and a dicer activator for use in the treatment of neuronal diseases
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
JP2021531038A (ja) * 2018-07-13 2021-11-18 ザ、トラスティーズ オブ プリンストン ユニバーシティ ストレス顆粒集合を調節するためのシステム及び方法
CA3112135A1 (en) * 2018-09-20 2020-03-26 Yeda Research And Development Co. Ltd. Methods of treating amyotrophic lateral sclerosis
CN113196055B (zh) * 2018-10-15 2025-06-27 马克斯·普朗克科学促进协会 用于治疗疾病的化合物及其筛选方法
WO2020086954A2 (en) * 2018-10-26 2020-04-30 The Jackson Laboratory Treatments for charcot-marie-tooth disease
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3921650A1 (en) 2019-02-08 2021-12-15 Dewpoint Therapeutics, Inc. Methods of characterizing condensate-associated characteristics of compounds and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020230133A1 (en) 2019-05-13 2020-11-19 Yeda Research And Development Co. Ltd. Cell-free mirna biomarkers for prognosis and diagnosis of neurodegenerative diseases
US11851462B2 (en) * 2019-07-22 2023-12-26 University Of South Carolina Targeting G3BP aggregation to prevent neurodegeneration
CN114729941A (zh) 2019-09-18 2022-07-08 露点治疗公司 筛选凝聚物相关特异性的方法及其用途
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021178448A1 (en) * 2020-03-02 2021-09-10 Motor Life Sciences, Llc Compositions and methods for diagnosing, preventing, and treating amyotrophic lateral sclerosis in patients with hypofunctional anti-trypsin activity
WO2021178344A1 (en) * 2020-03-04 2021-09-10 The Trustees Of Indiana University Methods to re-engage a fetal wound healing pathway for adult skin repair
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CN112043721B (zh) * 2020-09-04 2022-01-28 南通大学 miR-132-5p在制备神经再生药物或材料中的应用
KR20220035693A (ko) * 2020-09-14 2022-03-22 주식회사 제너로스 표적화된 유전자 전달을 위한 아데노-부속 바이러스 벡터
WO2024163383A2 (en) * 2023-01-30 2024-08-08 Academia Sinica Treating spinal muscular atrophy (sma) by modulating mir34 and use of mir34 as a predictive biomarker of sma
AU2024288170A1 (en) * 2023-07-07 2026-01-22 The Board Of Regents Of The University Of Texas System Targeting heterogeneous nuclear ribonucleoproteins for neuroprotection

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154600B (nl) 1971-02-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van specifiek bindende eiwitten en hun corresponderende bindbare stoffen.
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
NL154599B (nl) 1970-12-28 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van specifiek bindende eiwitten en hun corresponderende bindbare stoffen, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3853987A (en) 1971-09-01 1974-12-10 W Dreyer Immunological reagent and radioimmuno assay
US3867517A (en) 1971-12-21 1975-02-18 Abbott Lab Direct radioimmunoassay for antigens and their antibodies
NL171930C (nl) 1972-05-11 1983-06-01 Akzo Nv Werkwijze voor het aantonen en bepalen van haptenen, alsmede testverpakkingen.
US3850578A (en) 1973-03-12 1974-11-26 H Mcconnell Process for assaying for biologically active molecules
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
US4879219A (en) 1980-09-19 1989-11-07 General Hospital Corporation Immunoassay utilizing monoclonal high affinity IgM antibodies
US5011771A (en) 1984-04-12 1991-04-30 The General Hospital Corporation Multiepitopic immunometric assay
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5281521A (en) 1992-07-20 1994-01-25 The Trustees Of The University Of Pennsylvania Modified avidin-biotin technique
US6756369B2 (en) 1997-05-06 2004-06-29 Vanderbilt University Diagnosis and management of infection caused by Chlamydia
FR2801217B1 (fr) * 1999-11-24 2002-12-06 Aventis Pharma Sa Association de riluzole et de gabapentine et son utilisation comme medicament
JP2006519008A (ja) 2003-02-10 2006-08-24 独立行政法人産業技術総合研究所 哺乳動物細胞の調節
CN1475215A (zh) * 2003-05-16 2004-02-18 海口康力元制药有限公司 注射用依诺沙星及其制作方法
EP2302055B1 (en) 2004-11-12 2014-08-27 Asuragen, Inc. Methods and compositions involving miRNA and miRNA inhibitor molecules
EP1962861A2 (en) 2005-08-26 2008-09-03 Emory University Compounds and methods for modulating the silencing of a polynucleotide of interest
US20070197548A1 (en) * 2006-02-17 2007-08-23 Murthy Yerramilli V S Fluoroquinolone compositions
WO2008073961A2 (en) 2006-12-12 2008-06-19 Emory University Compounds and methods for modulating the silencing of a polynucleotide of interest
CN101390854A (zh) * 2007-09-21 2009-03-25 北京德众万全药物技术开发有限公司 一种含有利鲁唑的药用组合物
EP2265291B1 (en) 2008-03-17 2016-10-19 The Board of Regents of The University of Texas System Identification of micro-rnas involved in neuromuscular synapse maintenance and regeneration
WO2010006425A1 (en) 2008-07-14 2010-01-21 Delaney Technologies Inc. Deployment mechanism for a retractable roof system for a large building structure
ES2554765T3 (es) * 2008-12-05 2015-12-23 Yeda Research And Development Co. Ltd. miRNA-9 o miRNA-9* para uso en el tratamiento de ELA
US9867837B2 (en) * 2011-03-01 2018-01-16 Pharnext Compositions for treating neurological disorders
WO2012162249A1 (en) 2011-05-20 2012-11-29 Benjamin Wolozin Identification of compounds that disperse tdp-43 inclusions
US20140348857A1 (en) 2011-10-27 2014-11-27 Massachusetts Institute Of Technology Methods of diagnosis and treatment of endoplasmic reticulum (er) stress-related conditions
JP6340366B2 (ja) 2012-07-31 2018-06-06 イェダ リサーチ アンド デベロップメント カンパニー リミテッド 運動ニューロン疾患を診断および処置する方法

Similar Documents

Publication Publication Date Title
JP2015523410A5 (https=)
HK1205453A1 (en) Methods of diagnosing and treating motor neuron diseases
EP3406246A3 (en) Alpha7 beta 1 integrin modulators for treating muscular dystrophy
WO2012036433A3 (ko) miRNA를 타겟으로 한 신경퇴행성 질환 치료
WO2016210372A3 (en) Methods to treat neurological diseases
WO2012174338A3 (en) Method of selecting therapeutic indications
WO2015033319A3 (en) Apparatus and methods for diagnosis and treatment of patterns of nervous system activity affecting disease
CA2812940A1 (en) Compositions and methods useful for the treatment and diagnosis of inflammatory bowel disease
WO2014144606A3 (en) Methods of treating muscular dystrophy
WO2014022739A3 (en) Modified rnai agents
BR112014014262A2 (pt) nanopartículas; composição farmacêutica; método de administrar um ou mais agentes terapêuticos, profiláticos, e/ou diagnósticos a um paciente em necessidade dos mesmos e método de preparação das partículas.
MX360179B (es) Complejo nanomolecular nts-poliplex que comprende el gen bdnf, para usarse en el tratamiento de la enfermedad de parkinson y composiciones farmacéuticas que lo contienen.
WO2010144721A3 (en) Model systems and treatment regimes for treatment of neurological disease
WO2014043696A3 (en) Compositions and methods for treating amyotrophic lateral sclerosis
WO2010025321A3 (en) Method for treating multiple sclerosis patients with anti-il2r antibodies
HK1200274A1 (en) Single nucleotide polymorphisms useful to predict clinical response for glatiramer acetate
HK1213830A1 (zh) 用於预测醋酸格拉替雷临床反应的生物标志物
WO2013084236A8 (en) Diagnostic and therapeutic methods and their application in amyotrophic lateral sclerosis (als)
FR2917613B1 (fr) Composition cosmetique ou pharmaceutique comprenant un polycondensat, procede de traitement cosmetique, polycondensat et procede de preparation.
HK1207953A1 (en) An enhanced therapeutic stimulus system and methods of use
WO2013173364A3 (en) Lingo-2 antagonists for treatment of conditions involving motor neurons
WO2015081283A3 (en) Long non-coding rna as a diagnostic and therapeutic agent
WO2016030899A8 (en) Methods of treating amyotrophic lateral scleroses
WO2010064248A3 (en) Methods of diagnosing and treating motor neuron diseases
WO2015069883A3 (en) Oxidized fraction of extracellular dna as a biomarker of stress and methods for using the same