JPWO2021142342A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021142342A5
JPWO2021142342A5 JP2022542340A JP2022542340A JPWO2021142342A5 JP WO2021142342 A5 JPWO2021142342 A5 JP WO2021142342A5 JP 2022542340 A JP2022542340 A JP 2022542340A JP 2022542340 A JP2022542340 A JP 2022542340A JP WO2021142342 A5 JPWO2021142342 A5 JP WO2021142342A5
Authority
JP
Japan
Prior art keywords
sequence
improved
seq
protein
nucleic acid
Prior art date
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.)
Pending
Application number
JP2022542340A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023510352A (ja
JP2023510352A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/012804 external-priority patent/WO2021142342A1/en
Publication of JP2023510352A publication Critical patent/JP2023510352A/ja
Publication of JPWO2021142342A5 publication Critical patent/JPWO2021142342A5/ja
Publication of JP2023510352A5 publication Critical patent/JP2023510352A5/ja
Pending legal-status Critical Current

Links

JP2022542340A 2020-01-10 2021-01-08 Pcsk9の標的化のための組成物および方法 Pending JP2023510352A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062959685P 2020-01-10 2020-01-10
US62/959,685 2020-01-10
PCT/US2021/012804 WO2021142342A1 (en) 2020-01-10 2021-01-08 Compositions and methods for the targeting of pcsk9

Publications (3)

Publication Number Publication Date
JP2023510352A JP2023510352A (ja) 2023-03-13
JPWO2021142342A5 true JPWO2021142342A5 (https=) 2024-01-16
JP2023510352A5 JP2023510352A5 (https=) 2024-01-16

Family

ID=74505368

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022542340A Pending JP2023510352A (ja) 2020-01-10 2021-01-08 Pcsk9の標的化のための組成物および方法

Country Status (9)

Country Link
US (1) US20230167424A1 (https=)
EP (1) EP4087930A1 (https=)
JP (1) JP2023510352A (https=)
KR (1) KR20220125332A (https=)
CN (1) CN115427570A (https=)
AU (1) AU2021206270A1 (https=)
CA (1) CA3163714A1 (https=)
IL (1) IL294620A (https=)
WO (1) WO2021142342A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
US20250388885A1 (en) * 2022-03-11 2025-12-25 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
WO2023235818A2 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
US20250361525A1 (en) 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Mecp2発現を低減する化合物及び方法
TW202444906A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向pcsk9之組合物及方法
WO2024238723A1 (en) * 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
CN118086311B (zh) * 2023-05-25 2024-08-09 苏州时安生物技术有限公司 抑制PCSK9基因表达的siRNA、其缀合物和药物组合物及用途
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
CN116978457B (zh) * 2023-09-22 2023-12-22 成都斯马特科技有限公司 避免rna检测过程中假基因干扰的引物和探针组合及其设计方法
CN119506351B (zh) * 2024-11-20 2025-08-19 中国人民解放军陆军军医大学第二附属医院 一体化pcsk9腺嘌呤碱基编辑系统及应用

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
CA2999649A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
CA3038960A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
EP3559223A1 (en) * 2016-12-23 2019-10-30 President and Fellows of Harvard College Gene editing of pcsk9
EP3585894A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
US11578334B2 (en) * 2017-10-25 2023-02-14 Monsanto Technology Llc Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes
WO2019126762A2 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
PH12021553076A1 (en) * 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
CA3153700A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy

Similar Documents

Publication Publication Date Title
JPWO2021142342A5 (https=)
JP7493487B2 (ja) 免疫を工学操作するための方法および組成物
US20230203540A1 (en) Methods and compositions for nuclease-mediated targeted integration of transgenes into mammalian liver cells
AU2017324462B2 (en) Modulation of liver genes
US9255250B2 (en) Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
EP3080143B1 (en) Methods and compositions for treating hemophilia
EP2890780B1 (en) Methods and compositions for treatment of a genetic condition
JP7832262B2 (ja) 肝特異的ウイルスプロモーター及びその使用方法
AU2017379073B2 (en) Compositions and methods for treating alpha-1 antitrypsin deficiency
CN116836957A (zh) 用于核酸酶介导的基因组工程改造的递送方法和组合物
Marsh et al. Application of CRISPR-Cas9-mediated genome editing for the treatment of myotonic dystrophy type 1
CN118401658A (zh) 调节pcsk9的方法及其用途
JP7756639B2 (ja) X連鎖性若年網膜分離療法のためのcrisprおよびaav戦略
JP2020527030A (ja) 肝臓において目的のタンパク質を発現するためのプラットフォーム
CA3147643A1 (en) Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (hnf4.alpha.) gene expression
CN117230043B (zh) Cas13蛋白及其应用
KR20250053925A (ko) CRISPR-Cas13 시스템 및 이의 용도
CN113474454A (zh) 可控的基因组编辑系统
JP2022548320A (ja) アポリポタンパク質b(apob)遺伝子発現をモジュレートするための組成物および方法
CN114072518B (zh) 用于治疗地中海贫血或镰状细胞病的方法和组合物
JP2025514304A (ja) 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定
WO2025261493A1 (zh) 通过表观编辑靶向调节pcsk9的方法及其用途
HK40052425A (en) Controllable genome editing system
KR20260048590A (ko) 유전자 편집 시스템 및 그 용도
HK40113388A (zh) 调节pcsk9的方法及其用途