JP2022521432A5 - - Google Patents

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Publication number
JP2022521432A5
JP2022521432A5 JP2021549631A JP2021549631A JP2022521432A5 JP 2022521432 A5 JP2022521432 A5 JP 2022521432A5 JP 2021549631 A JP2021549631 A JP 2021549631A JP 2021549631 A JP2021549631 A JP 2021549631A JP 2022521432 A5 JP2022521432 A5 JP 2022521432A5
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JP
Japan
Prior art keywords
nucleic acid
recognition helix
dna
seq
optionally
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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.)
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JP2021549631A
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English (en)
Japanese (ja)
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JP7624728B2 (ja
JP2022521432A (ja
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Priority claimed from PCT/US2020/019546 external-priority patent/WO2020176426A1/en
Publication of JP2022521432A publication Critical patent/JP2022521432A/ja
Publication of JP2022521432A5 publication Critical patent/JP2022521432A5/ja
Priority to JP2024184019A priority Critical patent/JP2025003531A/ja
Application granted granted Critical
Publication of JP7624728B2 publication Critical patent/JP7624728B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021549631A 2019-02-25 2020-02-24 Dna結合ドメイントランスアクチベーター及びその使用 Active JP7624728B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024184019A JP2025003531A (ja) 2019-02-25 2024-10-18 Dna結合ドメイントランスアクチベーター及びその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962810005P 2019-02-25 2019-02-25
US62/810,005 2019-02-25
PCT/US2020/019546 WO2020176426A1 (en) 2019-02-25 2020-02-24 Dna-binding domain transactivators and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024184019A Division JP2025003531A (ja) 2019-02-25 2024-10-18 Dna結合ドメイントランスアクチベーター及びその使用

Publications (3)

Publication Number Publication Date
JP2022521432A JP2022521432A (ja) 2022-04-07
JP2022521432A5 true JP2022521432A5 (https=) 2023-03-03
JP7624728B2 JP7624728B2 (ja) 2025-01-31

Family

ID=72238705

Family Applications (2)

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JP2021549631A Active JP7624728B2 (ja) 2019-02-25 2020-02-24 Dna結合ドメイントランスアクチベーター及びその使用
JP2024184019A Pending JP2025003531A (ja) 2019-02-25 2024-10-18 Dna結合ドメイントランスアクチベーター及びその使用

Family Applications After (1)

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JP2024184019A Pending JP2025003531A (ja) 2019-02-25 2024-10-18 Dna結合ドメイントランスアクチベーター及びその使用

Country Status (14)

Country Link
US (1) US12583902B2 (https=)
EP (1) EP3931213A4 (https=)
JP (2) JP7624728B2 (https=)
KR (1) KR20210132109A (https=)
CN (1) CN113710693B (https=)
AU (2) AU2020228028A1 (https=)
BR (1) BR112021016294A2 (https=)
CA (1) CA3129757A1 (https=)
CL (1) CL2021002226A1 (https=)
CO (1) CO2021011722A2 (https=)
IL (1) IL285687A (https=)
MX (1) MX2021010169A (https=)
SG (1) SG11202108568YA (https=)
WO (1) WO2020176426A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランスアクチベーター及びその使用
WO2022020706A1 (en) * 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof
WO2023042104A1 (en) * 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
JP2024545923A (ja) * 2021-12-30 2024-12-13 リーゲル セラピューティクス,インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット1の発現を調節するための組成物、及びその使用
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6794136B1 (en) * 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US20030082561A1 (en) 2000-07-21 2003-05-01 Takashi Sera Zinc finger domain recognition code and uses thereof
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US20060182736A1 (en) 2003-06-10 2006-08-17 Kim Jin-Soo Transducible dna-binding proteins
WO2007061759A1 (en) 2005-11-18 2007-05-31 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Delayed expression vectors
AU2010273220B2 (en) 2009-07-13 2015-10-15 President And Fellows Of Harvard College Bifunctional stapled polypeptides and uses thereof
US9567573B2 (en) * 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
JP2018513681A (ja) 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP6853257B2 (ja) 2015-09-23 2021-03-31 サンガモ セラピューティクス, インコーポレイテッド Httリプレッサー及びその使用
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
GB201517227D0 (en) * 2015-09-29 2015-11-11 Univ Edinburgh Genetically-edited swine
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
GB2544270A (en) 2015-11-05 2017-05-17 Fundació Centre De Regulació Genòmica Nucleic acids, peptides and methods
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
JP2018088888A (ja) * 2016-12-06 2018-06-14 国立研究開発法人理化学研究所 Scn1a遺伝子の発現増強法
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
MX2019011772A (es) 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
CA3096407A1 (en) 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019224864A1 (ja) 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Scn1a遺伝子の発現増強法とそれによるドラベ症候群の治療法
WO2020097121A1 (en) 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランスアクチベーター及びその使用
PL3976798T3 (pl) 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
WO2022020706A1 (en) 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof

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