JP2024519469A - 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 - Google Patents

遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 Download PDF

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JP2024519469A
JP2024519469A JP2023566465A JP2023566465A JP2024519469A JP 2024519469 A JP2024519469 A JP 2024519469A JP 2023566465 A JP2023566465 A JP 2023566465A JP 2023566465 A JP2023566465 A JP 2023566465A JP 2024519469 A JP2024519469 A JP 2024519469A
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vector
gene
pcsk9
aav
sequence
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JP2024519469A5 (enExample
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ワン，リリー
ウイルソン，ジェームス・エム
トレティアコバ，アンナ
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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JP2023566465A 2021-04-27 2022-04-27 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法 Pending JP2024519469A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163180603P 2021-04-27 2021-04-27
US63/180,603 2021-04-27
US202163242474P 2021-09-09 2021-09-09
US63/242,474 2021-09-09
US202163244205P 2021-09-14 2021-09-14
US63/244,205 2021-09-14
US202263301933P 2022-01-21 2022-01-21
US63/301,933 2022-01-21
US202263331385P 2022-04-15 2022-04-15
US63/331,385 2022-04-15
PCT/US2022/026483 WO2022232232A1 (en) 2021-04-27 2022-04-27 Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders

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JP2024519469A true JP2024519469A (ja) 2024-05-14
JP2024519469A5 JP2024519469A5 (enExample) 2025-05-07

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AU (1) AU2022266662A1 (enExample)
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CO (1) CO2023016031A2 (enExample)
IL (1) IL307958A (enExample)
MX (1) MX2023012747A (enExample)
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EP4466367A4 (en) * 2022-01-21 2026-03-18 Univ Pennsylvania METHODS FOR THE TREATMENT OF ORNITHIN TRANSCARBAMYLASE (OTC) DEFICIENCY
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2024015972A2 (en) * 2022-07-14 2024-01-18 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders in adult patients
KR20260027890A (ko) * 2023-04-28 2026-03-03 오스페달레 산 라파엘 에스.알.엘. 벡터 생산
EP4735603A2 (en) * 2023-06-30 2026-05-06 nChroma Bio, Inc. Guide rna compositions
CN116790604B (zh) * 2023-08-18 2023-10-27 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途
WO2025217184A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of phenylketonuria (pku)
WO2025217186A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of citrullinemia type 1 (ctln1)

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JP2020517255A (ja) * 2017-04-21 2020-06-18 プレシジョン バイオサイエンシズ，インク． Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

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JP6851319B2 (ja) * 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11083799B2 (en) * 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
WO2018058064A1 (en) * 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
EP3714048B1 (en) * 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

Patent Citations (1)

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JP2020517255A (ja) * 2017-04-21 2020-06-18 プレシジョン バイオサイエンシズ，インク． Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

Non-Patent Citations (1)

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YANG YANG ET AL.: "A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice", NATURE BIOTECHNOLOGY, vol. 34, no. 3, JPN6026022866, 2016, pages 334 - 338, ISSN: 0005877133 *

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KR20240001708A (ko) 2024-01-03
CA3216285A1 (en) 2022-11-03
TW202304528A (zh) 2023-02-01
AU2022266662A9 (en) 2023-11-16
WO2022232232A1 (en) 2022-11-03
AU2022266662A1 (en) 2023-11-02
BR112023021129A2 (pt) 2023-12-12
US20240197916A1 (en) 2024-06-20
EP4330412A1 (en) 2024-03-06
IL307958A (en) 2023-12-01
EP4330412A4 (en) 2026-01-28
CO2023016031A2 (es) 2023-12-11

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