BR112018071439A2 - composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro - Google Patents

composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro

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Publication number
BR112018071439A2
BR112018071439A2 BR112018071439-9A BR112018071439A BR112018071439A2 BR 112018071439 A2 BR112018071439 A2 BR 112018071439A2 BR 112018071439 A BR112018071439 A BR 112018071439A BR 112018071439 A2 BR112018071439 A2 BR 112018071439A2
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BR
Brazil
Prior art keywords
compositions
methods
nucleotide repeats
transcription factor
diseases associated
Prior art date
Application number
BR112018071439-9A
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English (en)
Inventor
Michael Burns Sean
Andrew Murray Bradley
Beth Hesse Sarah
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Intellia 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.)
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Publication date
Application filed by Intellia Therapeutics, Inc. filed Critical Intellia Therapeutics, Inc.
Publication of BR112018071439A2 publication Critical patent/BR112018071439A2/pt

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    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
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    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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 [CRISPRs]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
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  • Wood Science & Technology (AREA)
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  • Optics & Photonics (AREA)
  • Psychology (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
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  • Microbiology (AREA)

Abstract

a presente invenção refere-se a composições e métodos para remoção por excisão de repetições de três nucleotídeos (tnrs) contidas no intron 3 de tcf4, tal como é visto em indivíduos portadores de distrofia endotelial da córnea ou distrofia de fuchs (fecd), colangite esclerosante primária (cep) e esquizofrenia. além disso, são descritas também composições compreendendo sequências-guia que têm como alvo a subunidade alfa 2 do colágeno viii para o tratamento de mutações nesta subunidade que podem contribuir para distrofia endotelial da córnea ou de fuchs (fecd).
BR112018071439-9A 2016-04-22 2017-04-21 composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro BR112018071439A2 (pt)

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 (1)

Publication Number Publication Date
BR112018071439A2 true BR112018071439A2 (pt) 2019-03-19

Family

ID=58692575

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018071439-9A BR112018071439A2 (pt) 2016-04-22 2017-04-21 composições e métodos para o tratamento de doenças associadas com repetições de três nucleotídeos no fator de transcrição quatro

Country Status (11)

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
EP3723813A4 (en) * 2017-12-15 2021-10-06 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
CN113557010A (zh) * 2019-02-28 2021-10-26 瑞泽恩制药公司 用于递送治疗剂的腺相关病毒载体
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
AU2021394998A1 (en) 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
WO2023086842A1 (en) * 2021-11-09 2023-05-19 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2013101711A1 (en) * 2011-12-30 2013-07-04 Mayo Foundation For Medical Education And Research Assessing likelihood of developing fuchs' corneal dystrophy
DK2800811T3 (en) * 2012-05-25 2017-07-17 Univ Vienna METHODS AND COMPOSITIONS FOR RNA DIRECTIVE TARGET DNA MODIFICATION AND FOR RNA DIRECTIVE MODULATION OF TRANSCRIPTION
EP2931899A1 (en) * 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
JP6352950B2 (ja) 2013-03-08 2018-07-04 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物
CA2930015A1 (en) * 2013-11-07 2015-05-14 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CA2945335A1 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
BR112017028201A2 (pt) * 2015-07-02 2018-08-28 Univ Johns Hopkins tratamentos com base em crisp/cas9

Also Published As

Publication number Publication date
MX2018012873A (es) 2019-08-05
CO2018012433A2 (es) 2019-04-30
JP2019515914A (ja) 2019-06-13
EP3445375A1 (en) 2019-02-27
CN109414450A (zh) 2019-03-01
WO2017185054A1 (en) 2017-10-26
KR20180134412A (ko) 2018-12-18
CA3021647A1 (en) 2017-10-26
US20190142972A1 (en) 2019-05-16
AU2017254718A1 (en) 2018-11-15

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