CA3044531A1 - Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing - Google Patents

Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing Download PDF

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Publication number
CA3044531A1
CA3044531A1 CA3044531A CA3044531A CA3044531A1 CA 3044531 A1 CA3044531 A1 CA 3044531A1 CA 3044531 A CA3044531 A CA 3044531A CA 3044531 A CA3044531 A CA 3044531A CA 3044531 A1 CA3044531 A1 CA 3044531A1
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exon
human
composition
promoter
sequence
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CA3044531A
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English (en)
French (fr)
Inventor
Yu Zhang
Chengzu LONG
Rhonda Bassel-Duby
Eric Olson
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University of Texas System
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University of Texas System
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Publication of CA3044531A1 publication Critical patent/CA3044531A1/en
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    • C12N15/102—Mutagenizing nucleic acids
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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    • 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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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 [CRISPR]
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    • C12N2320/00—Applications; Uses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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CA3044531A 2016-11-28 2017-11-28 Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing Abandoned CA3044531A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662426853P 2016-11-28 2016-11-28
US62/426,853 2016-11-28
PCT/US2017/063468 WO2018098480A1 (en) 2016-11-28 2017-11-28 Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing

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Publication Number Publication Date
CA3044531A1 true CA3044531A1 (en) 2018-05-31

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CA3044531A Abandoned CA3044531A1 (en) 2016-11-28 2017-11-28 Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing

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US (1) US20200046854A1 (enExample)
EP (1) EP3545090A1 (enExample)
JP (1) JP2019536782A (enExample)
CN (1) CN110382695A (enExample)
AU (1) AU2017364106A1 (enExample)
CA (1) CA3044531A1 (enExample)
MX (1) MX2019006157A (enExample)
WO (1) WO2018098480A1 (enExample)

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