JP2019524149A - 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 - Google Patents

一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Download PDF

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JP2019524149A
JP2019524149A JP2019510339A JP2019510339A JP2019524149A JP 2019524149 A JP2019524149 A JP 2019524149A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019524149 A JP2019524149 A JP 2019524149A
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sequence
seq
snp
mutation
pam
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JP2019524149A5 (enExample
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タラ ムーア
タラ ムーア
アンドリュー ネスビット
アンドリュー ネスビット
デイビッド コートニー
デイビッド コートニー
ケイティー クリスティー
ケイティー クリスティー
ジーン リー
ジーン リー
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Avellino Lab USA Inc
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Avellino Lab USA Inc
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Publication of JP2019524149A publication Critical patent/JP2019524149A/ja
Publication of JP2019524149A5 publication Critical patent/JP2019524149A5/ja
Priority to JP2021214009A priority Critical patent/JP2022046694A/ja
Priority to JP2024002942A priority patent/JP2024041905A/ja
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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
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
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    • A—HUMAN NECESSITIES
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JP2019510339A 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Pending JP2019524149A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021214009A JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662377586P 2016-08-20 2016-08-20
US62/377,586 2016-08-20
US201762462808P 2017-02-23 2017-02-23
US62/462,808 2017-02-23
US201762501750P 2017-05-05 2017-05-05
US62/501,750 2017-05-05
PCT/US2017/047861 WO2018039145A1 (en) 2016-08-20 2017-08-21 Single guide rna, crispr/cas9 systems, and methods of use thereof

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JP2021214009A Division JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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JP2019524149A true JP2019524149A (ja) 2019-09-05
JP2019524149A5 JP2019524149A5 (enExample) 2020-10-01

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JP2019510339A Pending JP2019524149A (ja) 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2021214009A Pending JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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JP2021214009A Pending JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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US (1) US20190185850A1 (enExample)
EP (1) EP3500677A4 (enExample)
JP (3) JP2019524149A (enExample)
KR (2) KR102594051B1 (enExample)
CN (1) CN109963945A (enExample)
WO (1) WO2018039145A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
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JPWO2018225807A1 (ja) * 2017-06-07 2020-04-09 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬
JP2022046694A (ja) * 2016-08-20 2022-03-23 アベリノ ラボ ユーエスエー インコーポレイテッド 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
US11987809B2 (en) 2015-11-13 2024-05-21 Avellino Lab Usa, Inc. Methods for the treatment of corneal dystrophies

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EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
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
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2020046861A1 (en) * 2018-08-27 2020-03-05 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
WO2019165322A1 (en) * 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
WO2020225754A1 (en) * 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
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