JP2022545950A5 - - Google Patents

Info

Publication number
JP2022545950A5
JP2022545950A5 JP2022513443A JP2022513443A JP2022545950A5 JP 2022545950 A5 JP2022545950 A5 JP 2022545950A5 JP 2022513443 A JP2022513443 A JP 2022513443A JP 2022513443 A JP2022513443 A JP 2022513443A JP 2022545950 A5 JP2022545950 A5 JP 2022545950A5
Authority
JP
Japan
Prior art keywords
tada
spcas9
variant
deaminase
sequence
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.)
Granted
Application number
JP2022513443A
Other languages
English (en)
Japanese (ja)
Other versions
JP7781742B2 (ja
JP2022545950A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/048510 external-priority patent/WO2021041885A2/en
Publication of JP2022545950A publication Critical patent/JP2022545950A/ja
Publication of JP2022545950A5 publication Critical patent/JP2022545950A5/ja
Priority to JP2025135451A priority Critical patent/JP2025183226A/ja
Application granted granted Critical
Publication of JP7781742B2 publication Critical patent/JP7781742B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022513443A 2019-08-29 2020-08-28 転写または発現を可能にするように変異を編集するための組成物および方法 Active JP7781742B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025135451A JP2025183226A (ja) 2019-08-29 2025-08-15 転写または発現を可能にするように変異を編集するための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962893638P 2019-08-29 2019-08-29
US62/893,638 2019-08-29
PCT/US2020/048510 WO2021041885A2 (en) 2019-08-29 2020-08-28 Compositions and methods for editing a mutation to permit transcription or expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025135451A Division JP2025183226A (ja) 2019-08-29 2025-08-15 転写または発現を可能にするように変異を編集するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2022545950A JP2022545950A (ja) 2022-11-01
JP2022545950A5 true JP2022545950A5 (enExample) 2023-09-05
JP7781742B2 JP7781742B2 (ja) 2025-12-08

Family

ID=74684607

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022513443A Active JP7781742B2 (ja) 2019-08-29 2020-08-28 転写または発現を可能にするように変異を編集するための組成物および方法
JP2025135451A Pending JP2025183226A (ja) 2019-08-29 2025-08-15 転写または発現を可能にするように変異を編集するための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025135451A Pending JP2025183226A (ja) 2019-08-29 2025-08-15 転写または発現を可能にするように変異を編集するための組成物および方法

Country Status (8)

Country Link
US (1) US20220313799A1 (enExample)
EP (1) EP4022050A4 (enExample)
JP (2) JP7781742B2 (enExample)
KR (1) KR20220066289A (enExample)
CN (2) CN119913134A (enExample)
AU (1) AU2020336953A1 (enExample)
CA (1) CA3152861A1 (enExample)
WO (1) WO2021041885A2 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN119913134A (zh) * 2019-08-29 2025-05-02 比姆医疗股份有限公司 用于编辑突变以允许转录或表达的组合物和方法
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
EP4309669A4 (en) * 2021-03-16 2025-09-24 Shanghai Iaso Biotechnology Co Ltd UNIVERSAL CHIMERIC ANTIGEN RECEPTOR T LYMPHOCYTE AND ITS APPLICATION
EP4323501A1 (en) * 2021-04-16 2024-02-21 Beam Therapeutics Inc. Genetic modification of hepatocytes
KR20240012377A (ko) * 2021-05-28 2024-01-29 빔 테라퓨틱스, 인크. 염기 편집기의 자기-불활성화용 조성물 및 방법
US20250223584A2 (en) 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
JP2025519070A (ja) 2022-05-17 2025-06-24 エンヴェロップ セラピューティクス, インコーポレイテッド 効率的in vivo送達のための組成物および方法
KR20230166378A (ko) 2022-05-30 2023-12-07 현대자동차주식회사 차량의 제동 방법 및 장치
WO2024138131A1 (en) * 2022-12-22 2024-06-27 Trustees Of Tufts College Expanding applications of zgtc alphabet in protein expression and gene editing
WO2025064656A1 (en) * 2023-09-19 2025-03-27 Beam Therapeutics Inc. Compositions and methods for altering a nucleobase in a lipoprotein(a) polynucleotide

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004020658A1 (en) * 2002-08-30 2004-03-11 The Hospital For Sick Children Diagnosis of shwachman-diamond syndrome
US20110151447A1 (en) * 2007-11-06 2011-06-23 Children's Medical Center Corporation Method to produce induced pluripotent stem (ips) cells from non-embryonic human cells
AU2015280069B2 (en) * 2014-06-23 2021-08-12 The General Hospital Corporation Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq)
AU2016226077B2 (en) * 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
WO2017070632A2 (en) * 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018119359A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2018251801B2 (en) * 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
US11371030B2 (en) * 2017-05-31 2022-06-28 The University Of Tokyo Modified Cas9 protein and use thereof
WO2019040650A1 (en) * 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
CA3082251A1 (en) * 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN109306361B (zh) * 2018-02-11 2022-06-28 华东师范大学 一种新的a/t到g/c碱基定点转换的基因编辑系统
WO2019217942A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
CN119913134A (zh) * 2019-08-29 2025-05-02 比姆医疗股份有限公司 用于编辑突变以允许转录或表达的组合物和方法

Similar Documents

Publication Publication Date Title
US20230018543A1 (en) Crispr/cas-mediated gene conversion
JP2022546608A5 (enExample)
JP7197363B2 (ja) ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集
JP2025183226A5 (enExample)
JP6170080B2 (ja) 異常ヘモグロビン症の治療のための組成物および方法
JP2024525665A (ja) 効率的なゲノム編集のための組成物及び方法
US20220305141A1 (en) Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators
CA3151279A1 (en) Highly efficient dna base editors mediated by rna-aptamer recruitment for targeted genome modification and uses thereof
AU2016381313A1 (en) Compositions and methods for the treatment of hemoglobinopathies
CN107532182A (zh) 治疗血红蛋白病的材料和方法
CN109295053A (zh) 通过诱导剪接位点碱基突变或多聚嘧啶区碱基置换调控rna剪接的方法
CN117242184A (zh) 向导RNA设计及用于V型Cas系统的复合物
JP2022532139A5 (enExample)
JP2023545497A5 (enExample)
Cortés-Mancera et al. Gene-targeted DNA methylation: towards long-lasting reprogramming of gene expression?
JP2022526669A (ja) ジストロフィン機能を修復するためのCRISPR/Casをベースにした塩基編集組成物
JP2024511621A5 (enExample)
JP2024546841A5 (enExample)
JP7736329B2 (ja) Dux4遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法
Romano et al. Characterization of five evolutionary conserved regions of the human tyrosine hydroxylase (TH) promoter: Implications for the engineering of a human TH minimal promoter assembled in a self‐inactivating lentiviral vector system
JP2024506645A (ja) 転写調節遺伝子の編集による疾患の治癒
US20240309347A1 (en) Fusion Proteins for CRISPR-based Transcriptional Repression
CN115873850A (zh) 腺嘌呤碱基编辑系统及其应用
WO2025036482A1 (en) Type ii cas protein, crispr-cas system and uses thereof
WO2019194320A1 (ja) エンジニアリングされたBlCas9ヌクレアーゼ