JP7642809B2 - ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ - Google Patents

ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ Download PDF

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JP7642809B2
JP7642809B2 JP2023528230A JP2023528230A JP7642809B2 JP 7642809 B2 JP7642809 B2 JP 7642809B2 JP 2023528230 A JP2023528230 A JP 2023528230A JP 2023528230 A JP2023528230 A JP 2023528230A JP 7642809 B2 JP7642809 B2 JP 7642809B2
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seq
acid sequence
dmd
nucleic acid
engineered meganuclease
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JP2023549357A (ja
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オーウェンズ，ゲイリー
ラペ，ジャネル
スミス，ジェームス，ジェファーソン
モリス，ジョン
ターナー，ケイトリン
ルイス，ホイットニー
ジャンツ，デレック
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プレシジョン バイオサイエンシズ，インク．
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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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JP2023528230A 2020-11-12 2021-11-12 ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ Active JP7642809B2 (ja)

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JP2025028775A JP2025098002A (ja) 2020-11-12 2025-02-26 ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ

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US202063113131P 2020-11-12 2020-11-12
US63/113,131 2020-11-12
US202163233664P 2021-08-16 2021-08-16
US63/233,664 2021-08-16
PCT/US2021/059146 WO2022104062A1 (en) 2020-11-12 2021-11-12 Engineered meganucleases having specificity for recognition sequences in the dystrophin gene

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US (4) US11753630B2 (https=)
EP (1) EP4244342B1 (https=)
JP (2) JP7642809B2 (https=)
KR (2) KR102826438B1 (https=)
CN (3) CN120098962A (https=)
CA (1) CA3172481A1 (https=)
IL (2) IL317538A (https=)
MX (1) MX2023005496A (https=)
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WO2024216116A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
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JP2017512767A (ja) 2014-03-12 2017-05-25 プレシジョン バイオサイエンシズ，インク． 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失

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WO2011141820A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
JP2017512767A (ja) 2014-03-12 2017-05-25 プレシジョン バイオサイエンシズ，インク． 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失

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CN120098963A (zh) 2025-06-06
IL302733B2 (en) 2025-05-01
CA3172481A1 (en) 2022-05-19
EP4244342A1 (en) 2023-09-20
KR20250099412A (ko) 2025-07-01
CN120098962A (zh) 2025-06-06
IL302733B1 (en) 2025-01-01
MX2023005496A (es) 2023-08-04
KR20230118841A (ko) 2023-08-14
US11753630B2 (en) 2023-09-12
IL302733A (en) 2023-07-01
EP4244342B1 (en) 2026-04-01
CN116783293A (zh) 2023-09-19
CN116783293B (zh) 2025-03-18
WO2022104062A1 (en) 2022-05-19
IL317538A (en) 2025-02-01
US20240018495A1 (en) 2024-01-18
JP2025098002A (ja) 2025-07-01
US11993793B2 (en) 2024-05-28
JP2023549357A (ja) 2023-11-24
US20240002823A1 (en) 2024-01-04
US12006522B2 (en) 2024-06-11
US20230053176A1 (en) 2023-02-16
US20250034536A1 (en) 2025-01-30
KR102826438B1 (ko) 2025-07-04

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