JP2019531763A5 - - Google Patents

Download PDF

Info

Publication number
JP2019531763A5
JP2019531763A5 JP2019533313A JP2019533313A JP2019531763A5 JP 2019531763 A5 JP2019531763 A5 JP 2019531763A5 JP 2019533313 A JP2019533313 A JP 2019533313A JP 2019533313 A JP2019533313 A JP 2019533313A JP 2019531763 A5 JP2019531763 A5 JP 2019531763A5
Authority
JP
Japan
Prior art keywords
item
sequence
site
anchor sequence
specific
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
JP2019533313A
Other languages
English (en)
Japanese (ja)
Other versions
JP7682604B2 (ja
JP2019531763A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/050553 external-priority patent/WO2018049073A1/en
Publication of JP2019531763A publication Critical patent/JP2019531763A/ja
Publication of JP2019531763A5 publication Critical patent/JP2019531763A5/ja
Priority to JP2023113050A priority Critical patent/JP2023119040A/ja
Application granted granted Critical
Publication of JP7682604B2 publication Critical patent/JP7682604B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019533313A 2016-09-07 2017-09-07 遺伝子発現をモジュレートするための方法および組成物 Active JP7682604B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023113050A JP2023119040A (ja) 2016-09-07 2023-07-10 遺伝子発現をモジュレートするための方法および組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662384603P 2016-09-07 2016-09-07
US62/384,603 2016-09-07
US201662416501P 2016-11-02 2016-11-02
US62/416,501 2016-11-02
US201662439327P 2016-12-27 2016-12-27
US62/439,327 2016-12-27
US201762542703P 2017-08-08 2017-08-08
US62/542,703 2017-08-08
PCT/US2017/050553 WO2018049073A1 (en) 2016-09-07 2017-09-07 Methods and compositions for modulating gene expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023113050A Division JP2023119040A (ja) 2016-09-07 2023-07-10 遺伝子発現をモジュレートするための方法および組成物

Publications (3)

Publication Number Publication Date
JP2019531763A JP2019531763A (ja) 2019-11-07
JP2019531763A5 true JP2019531763A5 (https=) 2020-10-22
JP7682604B2 JP7682604B2 (ja) 2025-05-26

Family

ID=61562240

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019533313A Active JP7682604B2 (ja) 2016-09-07 2017-09-07 遺伝子発現をモジュレートするための方法および組成物
JP2023113050A Pending JP2023119040A (ja) 2016-09-07 2023-07-10 遺伝子発現をモジュレートするための方法および組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023113050A Pending JP2023119040A (ja) 2016-09-07 2023-07-10 遺伝子発現をモジュレートするための方法および組成物

Country Status (7)

Country Link
US (5) US20190255106A1 (https=)
EP (1) EP3510152A4 (https=)
JP (2) JP7682604B2 (https=)
CN (1) CN109890962A (https=)
AU (2) AU2017324550B2 (https=)
CA (1) CA3035910A1 (https=)
WO (4) WO2018049079A1 (https=)

Families Citing this family (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
CA3034369A1 (en) 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US20200149039A1 (en) 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
DK3565891T3 (da) 2017-01-09 2023-07-24 Whitehead Inst Biomedical Res Fremgangsmåder til ændring af genekspression ved forstyrrelse af transkriptionsfaktor-multimere, der strukturerer regulatoriske sløjfer
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3790980A4 (en) * 2018-05-06 2022-03-23 Emendobio Inc. DIFFERENTIAL KNOCKOUT OF AN ALLEL OF A HETEROZYGOUS ELAN GENE
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
AU2019326408A1 (en) * 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
US20220380760A1 (en) * 2018-10-15 2022-12-01 Flagship Pioneering Innovations V, Inc. Disrupting genomic complex assembly in fusion genes
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
JP2022513159A (ja) * 2018-11-29 2022-02-07 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド Rnaを調節する方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
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
WO2020176553A1 (en) * 2019-02-25 2020-09-03 Sense Therapeutics Inc. Intracellular mutation targeting therapy
KR20210140819A (ko) * 2019-03-18 2021-11-23 사운드 애그리컬쳐 컴퍼니 식물에서 유전자 발현의 프로그래밍가능한 후성적 제어
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
PL3976798T3 (pl) * 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
CA3154759A1 (en) * 2019-09-23 2021-04-01 Flagship Pioneering Innovations V, Inc. Modulating genomic complexes
EP4048807A1 (en) * 2019-09-23 2022-08-31 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein b (apob) gene expression
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
WO2021061640A1 (en) * 2019-09-23 2021-04-01 Flagship Pioneering Innovations V, Inc. Compositions and methods for modulating genomic complex integrity index
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US12435334B2 (en) 2019-11-06 2025-10-07 Emendobio Inc. Differential knockout of an allele of a heterozygous ELANE gene-II
US12258599B2 (en) 2019-11-06 2025-03-25 Emendobio Inc. Method to inactivate a mutant allele of an ELANE gene
US20220387515A1 (en) * 2019-11-06 2022-12-08 Emendobio Inc. Differential knockout of an allele of a heterozygous elane gene using guides 21-30 nucleotides in length
EP4061968A4 (en) * 2019-11-22 2023-12-27 National University of Singapore Method and system for identifying and validating shared candidate antigens and shared antigen-specific t lymphocyte pairs
CN114981424A (zh) * 2020-01-17 2022-08-30 尼祖姆贝公司 在染色质靶点处dna链断裂的诱导
JPWO2021171688A1 (https=) * 2020-02-26 2021-09-02
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
CA3173528A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
WO2021216457A1 (en) * 2020-04-24 2021-10-28 Spinalcyte, Llc Treatment of frontotemporal dementia using fibroblasts and products thereof
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
KR20230079181A (ko) * 2020-09-29 2023-06-05 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 다중 유전자의 발현을 억제하는 조성물 및 방법
CN112301020B (zh) * 2020-10-19 2024-04-12 复旦大学附属肿瘤医院 Iii类脱氧核酶突变体及其制备方法与应用
CN114621959B (zh) * 2020-12-08 2023-05-26 中国科学院海洋研究所 一种编码牙鲆igf2可溶性蛋白的基因及蛋白重组表达方法与应用
KR20240011120A (ko) * 2020-12-22 2024-01-25 크로마 메디슨, 인크. 후성유전학적 편집을 위한 조성물 및 방법
EP4308153A4 (en) * 2021-03-16 2025-12-17 Massachusetts Gen Hospital Compositions and methods for treating diseases associated with nucleoside repeat expansion
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
US20230116385A1 (en) * 2021-08-30 2023-04-13 Sachi Bioworks Inc. Composition of a therapeutic oligomer
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
GB2625500B (en) 2021-09-21 2026-04-01 Scribe Therapeutics Inc Engineered CasX repressor systems
WO2023225349A2 (en) * 2022-05-20 2023-11-23 Helex Inc. Tissue specific methods and compositions for gene editing
WO2023250429A2 (en) * 2022-06-22 2023-12-28 Flagship Pioneering Innovations V, Inc. Combination therapies comprising myc modulation
AU2023286673A1 (en) * 2022-06-22 2024-12-12 Acuitas Therapeutics, Inc. Formulations for modulating myc expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238723A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
EP4713027A1 (en) 2023-05-16 2026-03-25 Omega Therapeutics, Inc. Methods and compositions for modulating methylation of a target gene
EP4716741A2 (en) 2023-05-23 2026-04-01 Flagship Labs 114, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
WO2025019742A1 (en) 2023-07-19 2025-01-23 Omega Therapeutics, Inc. Methods and compositions for modulating ctnnb1 expression
WO2025085423A1 (en) * 2023-10-19 2025-04-24 The Broad Institute, Inc. Method of multi-locus crispri targeting with a single truncated guide
CN118773200B (zh) * 2024-07-18 2025-06-10 华中科技大学同济医学院附属同济医院 一种ttc21b基因突变位点及其应用

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1622569B1 (en) 2003-04-24 2015-12-02 Incyte Corporation Aza spiro alkane derivatives as inhibitors of metallproteases
WO2009146033A2 (en) 2008-03-31 2009-12-03 Sma Foundation Compositions and methods for modulating smn activity
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
EP2414524B1 (en) 2009-04-03 2017-08-23 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2694686B2 (en) * 2011-04-06 2023-07-19 The University of Chicago COMPOSITION AND METHODS RELATED TO MODIFICATION OF 5-METHYLCYTOSINE (5mC)
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CN112301024A (zh) * 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
EP3636750A1 (en) 2013-09-04 2020-04-15 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9526784B2 (en) * 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015038892A1 (en) 2013-09-13 2015-03-19 Moderna Therapeutics, Inc. Polynucleotide compositions containing amino acids
WO2015191780A2 (en) * 2014-06-10 2015-12-17 The General Hospital Corporation Ccctc-binding factor (ctcf) rna interactome
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
WO2016014794A1 (en) * 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US10077453B2 (en) * 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
GB201418965D0 (https=) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
TWI716367B (zh) * 2014-10-31 2021-01-21 麻省理工學院 用於常間回文重複序列叢集(crispr)之大量平行組合性基因學
EP4464338A3 (en) * 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
EP3221813A4 (en) * 2014-11-20 2018-04-18 Children's Medical Center Corporation Methods relating to the detection of recurrent and non-specific double strand breaks in the genome
WO2016089920A1 (en) 2014-12-01 2016-06-09 The Broad Institute, Inc. Method for in situ determination of nucleic acid proximity
EP3237017A4 (en) 2014-12-24 2018-08-01 Dana-Farber Cancer Institute, Inc. Systems and methods for genome modification and regulation
CN107429263A (zh) * 2015-01-15 2017-12-01 斯坦福大学托管董事会 调控基因组编辑的方法
US10023922B2 (en) 2015-03-23 2018-07-17 Whitehead Institute For Biomedical Research Reporter of genomic methylation and uses thereof
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
EP3087974A1 (en) * 2015-04-29 2016-11-02 Rodos BioTarget GmbH Targeted nanocarriers for targeted drug delivery of gene therapeutics
US20170014449A1 (en) * 2015-07-13 2017-01-19 Elwha LLC, a limited liability company of the State of Delaware Site-specific epigenetic editing
WO2017011710A2 (en) 2015-07-14 2017-01-19 Whitehead Institute For Biomedical Research Chromosome neighborhood structures and methods relating thereto
WO2017031370A1 (en) * 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2017040793A1 (en) 2015-09-01 2017-03-09 Dana-Farber Cancer Institute Inc. Systems and methods for selection of grna targeting strands for cas9 localization
US20190048340A1 (en) * 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
US20170130247A1 (en) 2015-09-30 2017-05-11 Whitehead Institute For Biomedical Research Compositions and methods for altering gene expression
US20190062752A1 (en) 2015-10-29 2019-02-28 Whitehead Institute For Biomedical Research Transcription Factor Trapping by RNA in Gene Regulatory Elements
US11339442B2 (en) 2015-12-14 2022-05-24 The General Hospital Corporation Methods of detecting insulator dysfunction and oncogene activation for screening, diagnosis and treatment of patients in need thereof
JP2019508037A (ja) * 2016-02-16 2019-03-28 イェール ユニバーシティーYale Universit 標的化遺伝子編集を増強するための組成物およびその使用方法
GB201608000D0 (en) 2016-05-06 2016-06-22 Oxford Biodynamics Ltd Chromosome detection
CA3034369A1 (en) * 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
WO2018085460A2 (en) 2016-11-02 2018-05-11 Flagship Pioneering, Inc. Compositions and methods for cell delivery
US20200149039A1 (en) 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
DK3565891T3 (da) 2017-01-09 2023-07-24 Whitehead Inst Biomedical Res Fremgangsmåder til ændring af genekspression ved forstyrrelse af transkriptionsfaktor-multimere, der strukturerer regulatoriske sløjfer
US20210322577A1 (en) 2017-03-03 2021-10-21 Flagship Pioneering Innovations V, Inc. Methods and systems for modifying dna
WO2018204764A1 (en) 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
WO2019036430A1 (en) 2017-08-14 2019-02-21 Camp4 Therapeutics Corporation METHODS OF TREATING HEPATIC DISEASES
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
EP3587516B1 (en) 2018-06-27 2024-05-22 Ricoh Company, Ltd. Ink, method of manufacturing acrylic resin particle, printing method, ink accommodating unit, and inkjet printing device
US20220380760A1 (en) 2018-10-15 2022-12-01 Flagship Pioneering Innovations V, Inc. Disrupting genomic complex assembly in fusion genes
JP2022513159A (ja) 2018-11-29 2022-02-07 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド Rnaを調節する方法
WO2021061640A1 (en) 2019-09-23 2021-04-01 Flagship Pioneering Innovations V, Inc. Compositions and methods for modulating genomic complex integrity index
CA3154759A1 (en) 2019-09-23 2021-04-01 Flagship Pioneering Innovations V, Inc. Modulating genomic complexes
US20220348893A1 (en) 2019-09-23 2022-11-03 Omega Therapeutics, Inc Methods and compositions for modulating frataxin expression and treating friedrich's ataxia
AU2021350024A1 (en) 2020-09-24 2023-05-04 Flagship Pioneering Innovations V, Inc. Compositions and methods for inhibiting gene expression
KR20230079181A (ko) 2020-09-29 2023-06-05 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 다중 유전자의 발현을 억제하는 조성물 및 방법
CA3205133A1 (en) 2020-12-15 2022-06-23 Flagship Pioneering Innovations V, Inc. Compositions and methods for modulating myc expression

Similar Documents

Publication Publication Date Title
JP2019531763A5 (https=)
TWI880645B (zh) 抑制lpa之基因表現之組合物及方法
US11136597B2 (en) Compositions for enhancing targeted gene editing and methods of use thereof
US9828601B2 (en) Compositions for inhibiting checkpoint gene expression and uses thereof
US20210322577A1 (en) Methods and systems for modifying dna
JP2016513976A5 (https=)
US20200308590A1 (en) Compositions and methods for treatment of cystic fibrosis
TW201920227A (zh) 用於抑制脂蛋白元C-III (APOC3)表現之RNAi試劑及組合物
CA3035910A1 (en) Methods and compositions for modulating gene expression
US20180312839A1 (en) Methods and compositions for increasing smn expression
EP3573618B1 (en) Compositions and methods for hemoglobin production
JP2010515707A5 (https=)
CN104769112A (zh) 用于在细胞中表达蛋白质的方法和产品
EP4041894A1 (en) COMPOSITIONS AND METHODS FOR MODULATING HEPATOCYTE NUCLEAR FACTOR 4-ALPHA (HNF4a) GENE EXPRESSION
ES2291536T3 (es) Control de la expresion de gen usando un complejo de un oligonucleotido y un peptido regulatorio.
JP2022512739A (ja) 操作された長鎖散在反復エレメント(line)トランスポゾンおよびそれらの使用方法
WO2021183720A1 (en) Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
CA3097914A1 (en) Methods of gene therapy
Summerton Intracellular inactivation of specific nucleotide sequences: a general approach to the treatment of viral diseases and virally-mediated cancers
US20240279687A1 (en) Peptide nucleic acids for spatiotemporal control of crispr-cas binding
JP2020521491A5 (https=)
JP2024530169A (ja) 方法
US20220243211A1 (en) Peptide nucleic acid compositions with modified hoogsteen binding segments and methods of use thereof
US20220372474A1 (en) Hydroxymethyl-modified gamma-pna compositions and methods of use thereof
CN115317610A (zh) 基于vps9d1-as1靶点的aso药物在肿瘤治疗中的应用