SG11202105189RA - Nuclease-mediated repeat expansion - Google Patents

Nuclease-mediated repeat expansion

Info

Publication number
SG11202105189RA
SG11202105189RA SG11202105189RA SG11202105189RA SG11202105189RA SG 11202105189R A SG11202105189R A SG 11202105189RA SG 11202105189R A SG11202105189R A SG 11202105189RA SG 11202105189R A SG11202105189R A SG 11202105189RA SG 11202105189R A SG11202105189R A SG 11202105189RA
Authority
SG
Singapore
Prior art keywords
nuclease
mediated
repeat expansion
repeat
expansion
Prior art date
Application number
SG11202105189RA
Inventor
Daisuke Kajimura
Aarti Sharma-Kanning
Brittany Dubose
Gustavo Droguett
Chia-Jen Siao
Junko Kuno
David Frendewey
Brian Zambrowicz
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of SG11202105189RA publication Critical patent/SG11202105189RA/en

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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism

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  • Life Sciences & Earth Sciences (AREA)
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  • Medicinal Chemistry (AREA)
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  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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SG11202105189RA 2018-12-20 2019-12-13 Nuclease-mediated repeat expansion SG11202105189RA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862782461P 2018-12-20 2018-12-20
US201962829995P 2019-04-05 2019-04-05
PCT/US2019/066317 WO2020131632A1 (en) 2018-12-20 2019-12-13 Nuclease-mediated repeat expansion

Publications (1)

Publication Number Publication Date
SG11202105189RA true SG11202105189RA (en) 2021-06-29

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SG11202105189RA SG11202105189RA (en) 2018-12-20 2019-12-13 Nuclease-mediated repeat expansion

Country Status (12)

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US (2) US11690362B2 (en)
EP (1) EP3732291A1 (en)
JP (2) JP7449291B2 (en)
KR (1) KR20210105914A (en)
CN (2) CN116064550A (en)
AU (2) AU2019403015B2 (en)
BR (1) BR112021011703A2 (en)
CA (1) CA3120799A1 (en)
IL (2) IL301193A (en)
MX (1) MX2021007400A (en)
SG (1) SG11202105189RA (en)
WO (1) WO2020131632A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7449291B2 (en) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Nuclease-mediated repeat expansion
BR112023025224A2 (en) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc HUMAN CHROMOSOME 9 (C9ORF72) OPEN READING BOARD 72 IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN114736931A (en) * 2022-01-18 2022-07-12 赛业（苏州）生物科技有限公司 Method for knocking out mouse Abhd12 gene by using CRISPR/Cas9 system and application
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
CN114958852A (en) * 2022-06-09 2022-08-30 赛业（苏州）生物科技有限公司 Construction method and application of Ifnar1 gene knockout mouse animal model

Family Cites Families (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5523226A (en) 1993-05-14 1996-06-04 Biotechnology Research And Development Corp. Transgenic swine compositions and methods
US5670356A (en) 1994-12-12 1997-09-23 Promega Corporation Modified luciferase
US5874304A (en) 1996-01-18 1999-02-23 University Of Florida Research Foundation, Inc. Humanized green fluorescent protein genes and methods
AU8587598A (en) 1997-07-26 1999-02-16 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AU2884102A (en) 2000-12-07 2002-06-18 Sangamo Biosciences Inc Regulation of angiogenesis with zinc finger proteins
CA2435394C (en) 2001-01-22 2018-01-09 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
AU2002225187A1 (en) 2001-01-22 2002-07-30 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20060078552A1 (en) 2002-03-15 2006-04-13 Sylvain Arnould Hybrid and single chain meganucleases and use thereof
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
CA2497913C (en) 2002-09-05 2014-06-03 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
AU2003290518A1 (en) 2002-09-06 2004-04-23 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
DK2767161T3 (en) 2004-10-19 2018-05-07 Regeneron Pharma Method of generating an animal homozygous for genetic modification
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
AU2006224248B2 (en) 2005-03-15 2011-01-06 Cellectis I-Crei meganuclease variants with modified specificity, method of preparation and uses thereof
CN101117633B (en) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 Nucleus transplantation method
JP5632610B2 (en) 2006-12-14 2014-11-26 ダウ アグロサイエンシィズ エルエルシー Optimized non-canonical zinc finger protein
JP2008307007A (en) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag Human pluripotent stem cell induced from human tissue-originated undifferentiated stem cell after birth
DK3064599T3 (en) 2008-02-15 2019-04-08 Synthetic Genomics Inc METHODS IN VITRO FOR COMBINING AND COMBINATORY CONSTRUCTION OF NUCLEIC ACID MOLECULES
CN102037594A (en) 2008-04-11 2011-04-27 Utc电力公司 Fuel cell and bipolar plate having manifold sump
US8470594B2 (en) 2008-04-15 2013-06-25 President And Fellows Of Harvard College Methods for identifying agents that affect the survival of motor neurons
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
JP5932632B2 (en) 2009-03-20 2016-06-15 サンガモ バイオサイエンシーズ， インコーポレイテッド Modification of CXCR4 using modified zinc finger protein
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
EP2464219B1 (en) 2009-08-14 2019-10-23 Revivicor, Inc. Multi-transgenic pigs for diabetes treatment
US8518392B2 (en) 2009-08-14 2013-08-27 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
CA2779858C (en) 2009-10-29 2019-10-29 Aris N. Economides Multifunctional alleles
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
US9896729B2 (en) 2011-08-31 2018-02-20 The University Of Manchester Method for diagnosing a neurodegenerative disease
WO2013041577A1 (en) 2011-09-20 2013-03-28 Vib Vzw Methods for the diagnosis of amyotrophic lateral sclerosis and frontotemporal lobar degeneration
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
PT2800811T (en) 2012-05-25 2017-08-17 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014033644A2 (en) 2012-08-28 2014-03-06 Novartis Ag Methods of nuclease-based genetic engineering
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
DK2906696T4 (en) 2012-10-15 2023-02-27 Ionis Pharmaceuticals Inc METHODS OF MODULATING C9ORF72 EXPRESSION
CN110643600A (en) 2012-10-23 2020-01-03 基因工具股份有限公司 System for cutting target DNA and use thereof
ES2769310T3 (en) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modification and regulation of the genome based on CRISPR
MX2015007550A (en) 2012-12-12 2017-02-02 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications.
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
RU2690352C2 (en) 2013-02-20 2019-05-31 Регенерон Фармасютикалс, Инк. Genetic modification of rats
JP2016507244A (en) 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト（ゲーエムベーハー）Ｈｅｌｍｈｏｌｔｚ Ｚｅｎｔｒｕｍ ＭｕｅｎｃｈｅｎＤｅｕｔｓｃｈｅｓ Ｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ｆｕｅｒ Ｇｅｓｕｎｄｈｅｉｔ ｕｎｄ Ｕｍｗｅｌｔ （ＧｍｂＨ） Gene editing in oocytes by Cas9 nuclease
CN110540991B (en) 2013-03-15 2023-10-24 通用医疗公司 Enhancement of specificity of RNA-guided genome editing using truncated guide RNA (tru-gRNA)
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6411463B2 (en) 2013-04-16 2018-10-24 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Targeted modification of rat genome
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
KR102251168B1 (en) 2013-10-25 2021-05-13 셀렉티스 Design of rare-cutting endonucleases for efficient and specific targeting dna sequences comprising highly repetitive motives
RU2685914C1 (en) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Methods and compositions for genome targeted modification
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
WO2015143078A1 (en) 2014-03-18 2015-09-24 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015153760A2 (en) 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
EP3674408A1 (en) 2014-06-16 2020-07-01 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas
PT3155099T (en) 2014-06-23 2018-04-20 Regeneron Pharma Nuclease-mediated dna assembly
US20150376587A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
MX2016017317A (en) 2014-06-26 2017-08-24 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use.
US10342761B2 (en) 2014-07-16 2019-07-09 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
KR102531016B1 (en) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs
NZ731471A (en) 2014-12-05 2021-12-24 Regeneron Pharma Non-human animals having a humanized cluster of differentiation 47 gene
RU2707137C2 (en) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Methods and compositions for targeted genetic modification by single-step multiple targeting
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016174056A1 (en) 2015-04-27 2016-11-03 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
WO2016179112A1 (en) 2015-05-01 2016-11-10 Precision Biosciences, Inc. Precise deletion of chromoscomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
US10285388B2 (en) 2015-05-29 2019-05-14 Regeneron Pharmaceuticals, Inc. Non-human animals having a disruption in a C9ORF72 locus
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
US11390908B2 (en) 2015-09-02 2022-07-19 University Of Massachusetts Detection of gene loci with CRISPR arrayed repeats and/or polychromatic single guide ribonucleic acids
SG11201805320XA (en) * 2015-12-23 2018-07-30 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
JP7245651B2 (en) 2016-03-30 2023-03-24 インテリア セラピューティクス，インコーポレイテッド Lipid Nanoparticle Formulations for CRISPR/CAS Components
EP3442596A1 (en) 2016-04-14 2019-02-20 Université de Lausanne Treatment and/or prevention of dna-triplet repeat diseases or disorders
US20190270980A1 (en) 2016-07-25 2019-09-05 Mayo Foundation For Medical Education And Research Treating cancer
AU2017336100B2 (en) 2016-09-30 2023-11-30 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a C9ORF72 locus
CN110337493B (en) * 2016-10-28 2024-03-12 吉尼松公司 Compositions and methods for treating myotonic dystrophy
CA3039922A1 (en) 2016-10-28 2018-05-03 Genethon Compositions and methods for the treatment of myotonic 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
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
AU2018355343A1 (en) 2017-10-24 2020-05-07 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
JP7449291B2 (en) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Nuclease-mediated repeat expansion
WO2020219726A1 (en) 2019-04-23 2020-10-29 Sangamo Therapeutics, Inc. Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof

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