KR20210151916A - 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실 - Google Patents

뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실 Download PDF

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KR20210151916A
KR20210151916A KR1020217036783A KR20217036783A KR20210151916A KR 20210151916 A KR20210151916 A KR 20210151916A KR 1020217036783 A KR1020217036783 A KR 1020217036783A KR 20217036783 A KR20217036783 A KR 20217036783A KR 20210151916 A KR20210151916 A KR 20210151916A
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South Korea
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vector
seq
cas9
promoter
gene
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Korean (ko)
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찰스 에이. 게르스바흐
카렌 불락락
재클린 엔. 로빈슨-햄
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듀크 유니버시티
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    • 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
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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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    • C12N15/09—Recombinant DNA-technology
    • 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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    • C12N15/09—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
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    • C12N9/14—Hydrolases (3)
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020217036783A 2019-04-14 2020-04-14 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실 Pending KR20210151916A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962833760P 2019-04-14 2019-04-14
US62/833,760 2019-04-14
PCT/US2020/028148 WO2020214609A1 (en) 2019-04-14 2020-04-14 Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy

Publications (1)

Publication Number Publication Date
KR20210151916A true KR20210151916A (ko) 2021-12-14

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217036783A Pending KR20210151916A (ko) 2019-04-14 2020-04-14 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실

Country Status (17)

Country Link
US (1) US20220184229A1 (https=)
EP (1) EP3931331A4 (https=)
JP (2) JP2022529631A (https=)
KR (1) KR20210151916A (https=)
CN (1) CN113785063A (https=)
AR (1) AR118670A1 (https=)
AU (1) AU2020257898B2 (https=)
BR (1) BR112021020515A2 (https=)
CA (1) CA3137248A1 (https=)
CL (1) CL2021002680A1 (https=)
CO (1) CO2021013692A2 (https=)
EA (1) EA202192815A1 (https=)
IL (1) IL287163A (https=)
MX (1) MX2021012511A (https=)
SG (1) SG11202111172QA (https=)
TW (2) TWI887234B (https=)
WO (1) WO2020214609A1 (https=)

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WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US20230272428A1 (en) * 2019-12-16 2023-08-31 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
US20230346978A1 (en) * 2020-04-14 2023-11-02 University Of Massachusetts Dcas13-mediated therapeutic rna base editing for in vivo gene therapy
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
MX2024002988A (es) * 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024193704A1 (en) * 2023-03-22 2024-09-26 Huidagene Therapeutics Co., Ltd. Guide nucleic acids targeting dmd and uses thereof
WO2025221969A2 (en) 2024-04-18 2025-10-23 Sarepta Therapeutics, Inc. Genetic constructs for gene editing

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WO2005035718A2 (en) * 2003-10-03 2005-04-21 Welgen, Inc. Biderectional promoters for small rna expression
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
ES2788426T3 (es) * 2014-06-16 2020-10-21 Univ Johns Hopkins Composiciones y métodos para la expresión de ARNs de guía de CRISPR utilizando el promotor de H1
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
KR102787119B1 (ko) * 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
EP3199632A1 (en) * 2016-01-26 2017-08-02 ACIB GmbH Temperature-inducible crispr/cas system
WO2017139505A2 (en) * 2016-02-11 2017-08-17 The Regents Of The University Of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
US10687520B2 (en) * 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
US11779653B2 (en) * 2017-09-29 2023-10-10 The Regents Of The University Of California Multi-armed polyrotaxane platform for protected nucleic acid delivery

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Publication number Publication date
TW202104594A (zh) 2021-02-01
IL287163A (en) 2021-12-01
AR118670A1 (es) 2021-10-20
EP3931331A1 (en) 2022-01-05
CL2021002680A1 (es) 2022-07-15
EA202192815A1 (ru) 2022-01-24
CN113785063A (zh) 2021-12-10
US20220184229A1 (en) 2022-06-16
AU2020257898B2 (en) 2026-03-05
WO2020214609A1 (en) 2020-10-22
JP2022529631A (ja) 2022-06-23
AU2020257898A1 (en) 2021-11-04
BR112021020515A2 (pt) 2022-01-04
SG11202111172QA (en) 2021-11-29
CA3137248A1 (en) 2020-10-22
CO2021013692A2 (es) 2022-01-17
TW202536186A (zh) 2025-09-16
JP2025087680A (ja) 2025-06-10
TWI887234B (zh) 2025-06-21
MX2021012511A (es) 2022-01-06
EP3931331A4 (en) 2022-11-30

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