JP2019515914A - 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 - Google Patents

転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 Download PDF

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JP2019515914A
JP2019515914A JP2018555262A JP2018555262A JP2019515914A JP 2019515914 A JP2019515914 A JP 2019515914A JP 2018555262 A JP2018555262 A JP 2018555262A JP 2018555262 A JP2018555262 A JP 2018555262A JP 2019515914 A JP2019515914 A JP 2019515914A
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seq
nuclease
guide rna
grna
sequence
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JP2019515914A5 (enExample
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マイケル バーンズ，ショーン
マイケル バーンズ，ショーン
アンドリュー マレー，ブラッドリー
アンドリュー マレー，ブラッドリー
ベス ヘス，サラ
ベス ヘス，サラ
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インテリア セラピューティクス，インコーポレイテッド
インテリア セラピューティクス，インコーポレイテッド
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
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    • C12N15/102—Mutagenizing nucleic acids
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Immunology (AREA)
  • Optics & Photonics (AREA)
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  • Psychology (AREA)
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JP2018555262A 2016-04-22 2017-04-21 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 Pending JP2019515914A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662326700P 2016-04-22 2016-04-22
US62/326,700 2016-04-22
PCT/US2017/028981 WO2017185054A1 (en) 2016-04-22 2017-04-21 Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four

Publications (2)

Publication Number Publication Date
JP2019515914A true JP2019515914A (ja) 2019-06-13
JP2019515914A5 JP2019515914A5 (enExample) 2020-06-11

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JP2018555262A Pending JP2019515914A (ja) 2016-04-22 2017-04-21 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法

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US (2) US20190142972A1 (enExample)
EP (1) EP3445375A1 (enExample)
JP (1) JP2019515914A (enExample)
KR (1) KR20180134412A (enExample)
CN (1) CN109414450A (enExample)
AU (1) AU2017254718A1 (enExample)
BR (1) BR112018071439A2 (enExample)
CA (1) CA3021647A1 (enExample)
CO (1) CO2018012433A2 (enExample)
MX (1) MX2018012873A (enExample)
WO (1) WO2017185054A1 (enExample)

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BR112017028201A2 (pt) * 2015-07-02 2018-08-28 Univ Johns Hopkins tratamentos com base em crisp/cas9
WO2022072458A1 (en) * 2020-09-29 2022-04-07 Avellino Lab Usa, Inc. Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
BR112019012825A2 (pt) 2016-12-22 2019-11-26 Intellia Therapeutics Inc composições e métodos para tratar deficiência de alfa-1 antitripsina
US11512312B2 (en) 2017-03-10 2022-11-29 The Board Of Regents Of The University Of Texas System Treatment of Fuchs' endothelial corneal dystrophy
US20230201373A1 (en) * 2017-12-15 2023-06-29 Regents Of The University Of Minnesota Crispr-mediated genome editing with vectors
CN109929872A (zh) * 2017-12-18 2019-06-25 中国科学院遗传与发育生物学研究所 一种通过基因编辑技术创制番茄白果材料的方法
EP3728598A1 (en) * 2017-12-21 2020-10-28 Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by nhej
US20190151470A1 (en) * 2018-01-21 2019-05-23 RoverMed BioSciences, LLC Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes
TW202045186A (zh) * 2019-02-28 2020-12-16 弗里德里希 Ｅ 克魯斯 細胞外基質調節劑
EP3930694A1 (en) * 2019-02-28 2022-01-05 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
US20230349888A1 (en) * 2020-04-27 2023-11-02 Duke University A high-throughput screening method to discover optimal grna pairs for crispr-mediated exon deletion
CN111944814B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
CN111944813B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 核苷酸、病毒载体及其应用和RNAi药物制剂
JP2023553935A (ja) 2020-12-11 2023-12-26 インテリア セラピューティクス，インコーポレイテッド 脱アミノ化を伴うゲノム編集のためのポリヌクレオチド、組成物、及び方法
EP4288541A1 (en) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
US20250319202A1 (en) * 2021-11-09 2025-10-16 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
WO2024226536A1 (en) * 2023-04-24 2024-10-31 The General Hospital Corporation Methods and compositions for modifying genetic repeats
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

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WO2015089419A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
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WO2015070083A1 (en) * 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
WO2015089419A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
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Also Published As

Publication number Publication date
CN109414450A (zh) 2019-03-01
US20190142972A1 (en) 2019-05-16
US20250025580A1 (en) 2025-01-23
WO2017185054A1 (en) 2017-10-26
KR20180134412A (ko) 2018-12-18
AU2017254718A1 (en) 2018-11-15
BR112018071439A2 (pt) 2019-03-19
CA3021647A1 (en) 2017-10-26
MX2018012873A (es) 2019-08-05
CO2018012433A2 (es) 2019-04-30
EP3445375A1 (en) 2019-02-27

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