JP2022529065A - 効率的なｒｎａトランススプライシングのための三重らせんターミネータ - Google Patents

効率的なｒｎａトランススプライシングのための三重らせんターミネータ Download PDF

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JP2022529065A
JP2022529065A JP2021561943A JP2021561943A JP2022529065A JP 2022529065 A JP2022529065 A JP 2022529065A JP 2021561943 A JP2021561943 A JP 2021561943A JP 2021561943 A JP2021561943 A JP 2021561943A JP 2022529065 A JP2022529065 A JP 2022529065A
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splicing
nucleic acid
trans
sequence
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フィッシャー，クリシュナ・ジェイ
ベネット，ジャン
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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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    • C12N2320/33—Alteration of splicing
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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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Ophthalmology & Optometry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021561943A 2019-04-17 2020-04-17 効率的なｒｎａトランススプライシングのための三重らせんターミネータ Pending JP2022529065A (ja)

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JP2025077741A JP2025121988A (ja) 2019-04-17 2025-05-08 効率的なｒｎａトランススプライシングのための三重らせんターミネータ

Applications Claiming Priority (3)

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US201962835164P 2019-04-17 2019-04-17
US62/835,164 2019-04-17
PCT/US2020/028797 WO2020214973A1 (en) 2019-04-17 2020-04-17 Triple helix terminator for efficient rna trans-splicing

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JP2025077741A Division JP2025121988A (ja) 2019-04-17 2025-05-08 効率的なｒｎａトランススプライシングのための三重らせんターミネータ

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JP2022529065A true JP2022529065A (ja) 2022-06-16
JP2022529065A5 JP2022529065A5 (https=) 2023-04-25

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JP2025077741A Pending JP2025121988A (ja) 2019-04-17 2025-05-08 効率的なｒｎａトランススプライシングのための三重らせんターミネータ

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US (1) US20220204989A1 (https=)
EP (1) EP3956442A4 (https=)
JP (2) JP2022529065A (https=)
KR (1) KR20220002910A (https=)
CN (1) CN114040974B (https=)
AU (1) AU2020260154A1 (https=)
BR (1) BR112021020539A2 (https=)
CA (1) CA3133555A1 (https=)
IL (1) IL287243A (https=)
MX (1) MX2021012702A (https=)
WO (1) WO2020214973A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025121988A (ja) * 2019-04-17 2025-08-20 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 効率的なｒｎａトランススプライシングのための三重らせんターミネータ

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
WO2022220968A1 (en) * 2021-04-15 2022-10-20 U1 Bio, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
EP4511488A2 (en) * 2022-04-20 2025-02-26 Tacit Therapeutics, Inc. Stabilization of therapeutic trans-splicing rna molecules in human cells
WO2023215761A1 (en) * 2022-05-03 2023-11-09 Tacit Therapeutics, Inc. Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus
EP4522758A4 (en) * 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
WO2024112957A1 (en) 2022-11-23 2024-05-30 Amber Bio Inc. Gene-modifying endonucleases

Citations (6)

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Publication number Priority date Publication date Assignee Title
JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用
JP2015534811A (ja) * 2012-11-02 2015-12-07 バウアー，ヨハン 癌の治療に使用されるｒｎａトランススプライシング分子（ｒｔｍ）
JP2015536647A (ja) * 2012-10-16 2015-12-24 マサチューセッツ インスティテュート オブ テクノロジー 安定な非ポリアデニル化ｒｎａの生成
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017087900A1 (en) * 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules

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WO2019027869A1 (en) * 2017-07-31 2019-02-07 Massachusetts Institute Of Technology RNA-CLUSTER INDUCED TRANSCRIPT STABILIZER AND USES THEREOF
BR112021020539A2 (pt) * 2019-04-17 2022-01-04 Univ Pennsylvania Terminador de hélice tripla para trans-splicing de rna eficiente

Patent Citations (7)

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JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用
JP2015536647A (ja) * 2012-10-16 2015-12-24 マサチューセッツ インスティテュート オブ テクノロジー 安定な非ポリアデニル化ｒｎａの生成
JP2015534811A (ja) * 2012-11-02 2015-12-07 バウアー，ヨハン 癌の治療に使用されるｒｎａトランススプライシング分子（ｒｔｍ）
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017087900A1 (en) * 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
US20180369412A1 (en) * 2015-11-19 2018-12-27 The Trustees Of The University Of Pennsylvania Compositions and Methods for Correction of Heritable Ocular Disease
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules

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Title
GENES AND DEVELOPMENT, vol. 26, JPN6024011990, 2012, pages 2392 - 2407, ISSN: 0005296936 *
MOLECULAR THERAPY: NUCLEIC ACIDS, vol. 12, JPN6024011989, 2018, pages 294 - 308, ISSN: 0005296937 *
PNAS, vol. 109, JPN6024011991, 2012, pages 19202 - 19207, ISSN: 0005296935 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025121988A (ja) * 2019-04-17 2025-08-20 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 効率的なｒｎａトランススプライシングのための三重らせんターミネータ

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US20220204989A1 (en) 2022-06-30
WO2020214973A1 (en) 2020-10-22
CA3133555A1 (en) 2020-10-22
JP2025121988A (ja) 2025-08-20
MX2021012702A (es) 2022-01-24
AU2020260154A1 (en) 2021-11-11
CN114040974B (zh) 2025-11-04
CN114040974A (zh) 2022-02-11
EP3956442A4 (en) 2023-01-25
IL287243A (en) 2021-12-01
BR112021020539A2 (pt) 2022-01-04
KR20220002910A (ko) 2022-01-07
EP3956442A1 (en) 2022-02-23

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