JP2022529631A5 - - Google Patents

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Publication number
JP2022529631A5
JP2022529631A5 JP2021560849A JP2021560849A JP2022529631A5 JP 2022529631 A5 JP2022529631 A5 JP 2022529631A5 JP 2021560849 A JP2021560849 A JP 2021560849A JP 2021560849 A JP2021560849 A JP 2021560849A JP 2022529631 A5 JP2022529631 A5 JP 2022529631A5
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JP
Japan
Prior art keywords
grna
vector
promoter
cas9 protein
cas9
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2021560849A
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English (en)
Japanese (ja)
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JP2022529631A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/028148 external-priority patent/WO2020214609A1/en
Publication of JP2022529631A publication Critical patent/JP2022529631A/ja
Publication of JP2022529631A5 publication Critical patent/JP2022529631A5/ja
Priority to JP2025014814A priority Critical patent/JP2025087680A/ja
Pending legal-status Critical Current

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JP2021560849A 2019-04-14 2020-04-14 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失 Pending JP2022529631A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025014814A JP2025087680A (ja) 2019-04-14 2025-01-31 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025014814A Division JP2025087680A (ja) 2019-04-14 2025-01-31 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失

Publications (2)

Publication Number Publication Date
JP2022529631A JP2022529631A (ja) 2022-06-23
JP2022529631A5 true JP2022529631A5 (enExample) 2023-04-21

Family

ID=72837591

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021560849A Pending JP2022529631A (ja) 2019-04-14 2020-04-14 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失
JP2025014814A Pending JP2025087680A (ja) 2019-04-14 2025-01-31 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025014814A Pending JP2025087680A (ja) 2019-04-14 2025-01-31 デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのaavベクター媒介欠失

Country Status (17)

Country Link
US (1) US12629428B2 (enExample)
EP (1) EP3931331A4 (enExample)
JP (2) JP2022529631A (enExample)
KR (1) KR20210151916A (enExample)
CN (1) CN113785063A (enExample)
AR (1) AR118670A1 (enExample)
AU (2) AU2020257898B2 (enExample)
BR (1) BR112021020515A2 (enExample)
CA (1) CA3137248A1 (enExample)
CL (1) CL2021002680A1 (enExample)
CO (1) CO2021013692A2 (enExample)
EA (1) EA202192815A1 (enExample)
IL (1) IL287163A (enExample)
MX (1) MX2021012511A (enExample)
SG (1) SG11202111172QA (enExample)
TW (1) TWI887234B (enExample)
WO (1) WO2020214609A1 (enExample)

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EP2841572B1 (en) 2012-04-27 2019-06-19 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
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD 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
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
JP2023507174A (ja) * 2019-12-16 2023-02-21 プレジデント アンド フェローズ オブ ハーバード カレッジ Dmd変異の修正のための方法及び組成物
EP4136236A4 (en) * 2020-04-14 2024-06-26 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
WO2023039447A2 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
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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