JP2024138268A5 - - Google Patents

Download PDF

Info

Publication number
JP2024138268A5
JP2024138268A5 JP2024095566A JP2024095566A JP2024138268A5 JP 2024138268 A5 JP2024138268 A5 JP 2024138268A5 JP 2024095566 A JP2024095566 A JP 2024095566A JP 2024095566 A JP2024095566 A JP 2024095566A JP 2024138268 A5 JP2024138268 A5 JP 2024138268A5
Authority
JP
Japan
Prior art keywords
amino acid
rtt
cell
modification
domain
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
JP2024095566A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024138268A (ja
Filing date
Publication date
Priority claimed from PCT/US2019/031899 external-priority patent/WO2019217944A1/en
Application filed filed Critical
Publication of JP2024138268A publication Critical patent/JP2024138268A/ja
Publication of JP2024138268A5 publication Critical patent/JP2024138268A5/ja
Pending legal-status Critical Current

Links

JP2024095566A 2018-05-11 2024-06-13 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 Pending JP2024138268A (ja)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201862670588P 2018-05-11 2018-05-11
US62/670,588 2018-05-11
US201862780838P 2018-12-17 2018-12-17
US62/780,838 2018-12-17
US201962817986P 2019-03-13 2019-03-13
US62/817,986 2019-03-13
PCT/US2019/031899 WO2019217944A1 (en) 2018-05-11 2019-05-11 Methods of editing single nucleotide polymorphism using programmable base editor systems
JP2021513765A JP7596259B2 (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021513765A Division JP7596259B2 (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法

Publications (2)

Publication Number Publication Date
JP2024138268A JP2024138268A (ja) 2024-10-08
JP2024138268A5 true JP2024138268A5 (enExample) 2025-04-25

Family

ID=68466849

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2021513765A Active JP7596259B2 (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法
JP2021513764A Pending JP2021523738A (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法
JP2024095566A Pending JP2024138268A (ja) 2018-05-11 2024-06-13 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法
JP2024095618A Pending JP2024147545A (ja) 2018-05-11 2024-06-13 プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2021513765A Active JP7596259B2 (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法
JP2021513764A Pending JP2021523738A (ja) 2018-05-11 2019-05-11 プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024095618A Pending JP2024147545A (ja) 2018-05-11 2024-06-13 プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法

Country Status (8)

Country Link
US (2) US20210380955A1 (enExample)
EP (2) EP3790595A4 (enExample)
JP (4) JP7596259B2 (enExample)
KR (3) KR20210023832A (enExample)
CN (3) CN119587720A (enExample)
AU (2) AU2019266326A1 (enExample)
CA (2) CA3100037A1 (enExample)
WO (2) WO2019217943A1 (enExample)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CN118416088A (zh) 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
EP3633034A4 (en) 2017-05-31 2021-03-24 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
EP3720507A4 (en) * 2017-12-07 2021-08-25 Ramot at Tel-Aviv University Ltd. TREATMENT OF PARKINSON PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
CN112041444A (zh) 2018-03-14 2020-12-04 阿伯生物技术公司 新型crispr dna靶向酶及系统
JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク. プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
WO2019226953A1 (en) * 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN116497067B (zh) 2019-02-13 2025-01-24 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020236936A1 (en) * 2019-05-21 2020-11-26 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems
JP2022536364A (ja) 2019-06-13 2022-08-15 ザ ジェネラル ホスピタル コーポレイション 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法
JP7500927B2 (ja) * 2019-08-06 2024-06-18 日本製紙株式会社 プロテイン用マスキング剤
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS Therapeutic editing
US20230054569A1 (en) * 2019-12-18 2023-02-23 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
US12264341B2 (en) * 2020-01-24 2025-04-01 The General Hospital Corporation CRISPR-Cas enzymes with enhanced on-target activity
US12312613B2 (en) 2020-01-24 2025-05-27 The General Hospital Corporation Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
WO2021155166A1 (en) * 2020-01-31 2021-08-05 University Of Massachusetts A cas9-pdbd base editor platform with improved targeting range and specificity
WO2021163492A1 (en) * 2020-02-14 2021-08-19 Ohio State Innovation Foundation Nucleobase editors and methods of use thereof
IT202000008014A1 (it) * 2020-04-15 2021-10-15 Fond Telethon RNA guida e loro usi
US20230165909A1 (en) * 2020-04-21 2023-06-01 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
US20250263752A1 (en) * 2020-04-21 2025-08-21 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
US20230159913A1 (en) 2020-04-28 2023-05-25 The Broad Institute, Inc. Targeted base editing of the ush2a gene
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022015856A1 (en) * 2020-07-14 2022-01-20 The Regents Of The University Of California Compositions and methods for treating an inherited retinal disease
WO2022020800A2 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
WO2022027035A1 (en) * 2020-07-27 2022-02-03 The Children's Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
JP2023543803A (ja) * 2020-09-24 2023-10-18 ザ ブロード インスティテュート,インコーポレーテッド プライム編集ガイドrna、その組成物、及びその使用方法
CA3200521A1 (en) * 2020-11-06 2022-05-12 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
AU2021396392A1 (en) * 2020-12-10 2023-07-06 Apellis Pharmaceuticals, Inc. Genomic editing of complement
WO2022197727A1 (en) * 2021-03-15 2022-09-22 Duke University Generation of novel crispr genome editing agents using combinatorial chemistry
WO2022241270A2 (en) * 2021-05-14 2022-11-17 Beam Therapeutics Inc. Compositions and methods for treating transthyretin amyloidosis
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
KR20240012377A (ko) * 2021-05-28 2024-01-29 빔 테라퓨틱스, 인크. 염기 편집기의 자기-불활성화용 조성물 및 방법
WO2023064858A1 (en) * 2021-10-13 2023-04-20 Apellis Pharmaceuticals, Inc. Compositions and methods for genome editing the neonatal fc receptor
JP2024544013A (ja) 2021-12-03 2024-11-26 ザ ブロード インスティテュート,インコーポレーテッド 有効なin vivo送達のための組成物および方法
US20250154479A1 (en) * 2022-01-24 2025-05-15 Toolgen Incorporated Streptococcus pyogenes-derived cas9 variant
CN114480445B (zh) * 2022-01-26 2023-06-27 西南交通大学 一种人源超氧化物歧化酶hSOD1突变体的制备及其应用
WO2023217280A1 (en) * 2022-05-13 2023-11-16 Huidagene Therapeutics Co., Ltd. Programmable adenine base editor and uses thereof
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
CN115148281B (zh) * 2022-06-29 2023-07-14 广州源井生物科技有限公司 一种基因编辑点突变方案自动设计方法及系统
CN120225674A (zh) * 2022-09-08 2025-06-27 爱丁堡大学董事会 雷特综合征疗法
WO2024107784A2 (en) * 2022-11-15 2024-05-23 Vanderbilt University Repair of disease-associated single nucleotide variants via interallelic gene conversion
WO2024259364A2 (en) * 2023-06-15 2024-12-19 Beam Therapeutics Inc. Methods and compositions for treating alpha-1 antitrypsin deficiency
WO2025024486A2 (en) * 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
US20250059567A1 (en) * 2023-08-17 2025-02-20 The Trustees Of The University Of Pennsylvania Compositions and methods for the management and treatment of phenylketonuria
WO2025059520A1 (en) * 2023-09-15 2025-03-20 Beam Therapeutics Inc. Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide
WO2025061198A1 (zh) * 2023-09-22 2025-03-27 北京大学现代农业研究院 改进的基因组编辑方法
WO2025122725A1 (en) * 2023-12-06 2025-06-12 The Broad Institute, Inc. Methods and compositions for base editing of tpp1 in the treatment of batten disease
CN118120704B (zh) * 2024-01-30 2025-10-24 江苏集萃药康生物科技股份有限公司 一种免高脂饮食诱导的动脉粥样硬化小鼠模型的构建方法

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
EP1235914A2 (en) 1999-11-24 2002-09-04 Joseph Rosenecker Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
DE60023936T2 (de) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
SG10201912571XA (en) 2009-11-02 2020-02-27 Univ Washington Therapeutic nuclease compositions and methods
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
IL289736B2 (en) * 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
CA2951707A1 (en) * 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
WO2016141224A1 (en) * 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
US11512311B2 (en) * 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
KR102424476B1 (ko) * 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
US11286478B2 (en) * 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CA3026110A1 (en) * 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
KR20250103795A (ko) * 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
CN107043779B (zh) * 2016-12-01 2020-05-12 中国农业科学院作物科学研究所 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도

Similar Documents

Publication Publication Date Title
JP2024138268A5 (enExample)
JP7698828B2 (ja) Rnaを編集する方法および組成物
JPWO2019217944A5 (enExample)
JP7252328B2 (ja) Rnaを編集する方法および組成物
US20230018543A1 (en) Crispr/cas-mediated gene conversion
Hermant et al. TFs for TEs: the transcription factor repertoire of mammalian transposable elements
Li et al. Genome-wide analyses reveal a role of Polycomb in promoting hypomethylation of DNA methylation valleys
JP2025026840A5 (enExample)
EP3371306B1 (en) Materials and methods for treatment of hemoglobinopathies
AU2018352592C1 (en) Uses of adenosine base editors
Liang et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
JP2024038327A5 (enExample)
CN107532182A (zh) 治疗血红蛋白病的材料和方法
US20140329697A1 (en) Constructing method of high-throughput sequencing library and use thereof
CN108351350A (zh) 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
JPWO2021050571A5 (enExample)
JP2025102768A5 (enExample)
Zhang et al. Comprehensive off-target analysis of dCas9-SAM-mediated HIV reactivation via long noncoding RNA and mRNA profiling
EP3645721A1 (en) Methods for the treatment of disease with gene editing systems
Breunig et al. CRISPR tools for physiology and cell state changes: potential of transcriptional engineering and epigenome editing
Badat et al. Direct correction of haemoglobin E β-thalassaemia using base editors
Shimosuga et al. Locus-specific hypomethylation of the mouse IAP retrotransposon is associated with transcription factor-binding sites
CA3136700A1 (en) Gene-editing systems for modifying a scn9a or scn10a gene and methods of use thereof
WO2024240223A1 (en) Deaminases and variants thereof for use in base editing
WO2020167795A1 (en) Methods for targeted depletion of nucleic acids