MX2019006157A - Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. - Google Patents

Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.

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Publication number
MX2019006157A
MX2019006157A MX2019006157A MX2019006157A MX2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A
Authority
MX
Mexico
Prior art keywords
editing
myo
dystrophin
mediated
mutations
Prior art date
Application number
MX2019006157A
Other languages
English (en)
Spanish (es)
Inventor
Zhang Yu
Long Chengzu
BASSEL-DUBY Rhonda
Olson Eric
Original Assignee
Univ Texas
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 Univ Texas filed Critical Univ Texas
Publication of MX2019006157A publication Critical patent/MX2019006157A/es

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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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • 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
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    • 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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    • 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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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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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MX2019006157A 2016-11-28 2017-11-28 Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. MX2019006157A (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX2019006157A true MX2019006157A (es) 2019-11-21

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MX2019006157A MX2019006157A (es) 2016-11-28 2017-11-28 Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.

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Country Link
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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