JP2016533322A5 - - Google Patents

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Publication number
JP2016533322A5
JP2016533322A5 JP2016516540A JP2016516540A JP2016533322A5 JP 2016533322 A5 JP2016533322 A5 JP 2016533322A5 JP 2016516540 A JP2016516540 A JP 2016516540A JP 2016516540 A JP2016516540 A JP 2016516540A JP 2016533322 A5 JP2016533322 A5 JP 2016533322A5
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Japan
Prior art keywords
silica coating
polymer bound
arginine
decrease
growth factor
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JP2016516540A
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English (en)
Japanese (ja)
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JP2016533322A (ja
JP6595459B2 (ja
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Priority claimed from PCT/US2014/057000 external-priority patent/WO2015042585A1/en
Publication of JP2016533322A publication Critical patent/JP2016533322A/ja
Publication of JP2016533322A5 publication Critical patent/JP2016533322A5/ja
Application granted granted Critical
Publication of JP6595459B2 publication Critical patent/JP6595459B2/ja
Expired - Fee Related legal-status Critical Current
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JP2016516540A 2013-09-23 2014-09-23 種々の細胞集団におけるナノ粒子媒介遺伝子送達、ゲノム編集およびリガンド標的化修飾 Expired - Fee Related JP6595459B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361881072P 2013-09-23 2013-09-23
US61/881,072 2013-09-23
PCT/US2014/057000 WO2015042585A1 (en) 2013-09-23 2014-09-23 Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations

Publications (3)

Publication Number Publication Date
JP2016533322A JP2016533322A (ja) 2016-10-27
JP2016533322A5 true JP2016533322A5 (enExample) 2017-10-12
JP6595459B2 JP6595459B2 (ja) 2019-10-23

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016516540A Expired - Fee Related JP6595459B2 (ja) 2013-09-23 2014-09-23 種々の細胞集団におけるナノ粒子媒介遺伝子送達、ゲノム編集およびリガンド標的化修飾

Country Status (12)

Country Link
US (2) US10526616B2 (enExample)
EP (2) EP3520822A1 (enExample)
JP (1) JP6595459B2 (enExample)
KR (1) KR20160060133A (enExample)
CN (1) CN105579068A (enExample)
AU (1) AU2014321215B2 (enExample)
CA (2) CA2924535C (enExample)
ES (1) ES2719112T3 (enExample)
IL (1) IL244585B (enExample)
PT (1) PT3049116T (enExample)
RU (1) RU2670512C2 (enExample)
WO (1) WO2015042585A1 (enExample)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP4166161B1 (en) 2016-04-14 2026-03-04 Fred Hutchinson Cancer Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
RU2019121801A (ru) * 2016-12-14 2021-01-15 Лигандал, Инк. Композиции и способы для доставки полезной нагрузки в виде нуклеиновой кислоты и/или белка
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018129270A1 (en) 2017-01-05 2018-07-12 Fred Hutchinson Cancer Research Center Systems and methods to improve vaccine efficacy
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
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
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019143948A1 (en) 2018-01-18 2019-07-25 Fred Hutchinson Cancer Research Center Altering inflammatory states of immune cells in vivo by modulating cellular activation states
US20210148918A1 (en) * 2018-04-17 2021-05-20 Purdue Research Foundation Modifying surface of a live cell and the uses thereof
US20200208177A1 (en) * 2018-04-18 2020-07-02 Ligandal, Inc. Methods and compositions for genome editing
US20200149070A1 (en) * 2018-04-24 2020-05-14 Ligandal, Inc. Methods and compositions for genome editing
CN121136065A (zh) * 2018-04-27 2025-12-16 基因编辑有限公司 阳离子聚合物和用于生物分子递送的用途
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
JP7467458B2 (ja) * 2018-07-30 2024-04-15 中央研究院 コアセルベート複合化による治療用ナノ粒子および細菌を処理するためのそれらの使用
EP3856759A4 (en) 2018-09-25 2022-10-12 The University of Tokyo AMPHIPHILIC POLY(AMINO ACID), BLOCK COPOLYMER USING THE AMPHIPHIL POLYAMINO ACID AND COMPLEX WITH THE AMPHIPHILIC POLY(AMINO ACID) OR THE BLOCK COPOLYMER AND NUCLEIC ACID
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220296683A1 (en) * 2019-09-20 2022-09-22 University College Dublin Oral delivery system
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
KR20250022763A (ko) 2022-06-10 2025-02-17 캠프4 테라퓨틱스 코포레이션 조절 rna를 표적으로 하는 안티센스 올리고뉴클레오타이드를 사용하여 프로그래뉼린 발현을 조절하는 방법
EP4627084A1 (en) 2022-12-01 2025-10-08 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP4743577A1 (en) 2023-07-13 2026-05-20 Korro Bio, Inc. RNA EDITING OLIGONUCLEOTIDES AND THEIR USES
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2026136379A1 (en) 2024-12-17 2026-06-25 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2026151811A1 (en) 2025-01-07 2026-07-16 Camp4 Therapeutics Corporation Compositions and methods for treating urea cycle disorders

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US6379966B2 (en) 1999-02-26 2002-04-30 Mirus Corporation Intravascular delivery of non-viral nucleic acid
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
RU2295954C2 (ru) 2000-09-28 2007-03-27 Чирон Корпорейшн Микрочастицы для доставки гетерологичных нуклеиновых кислот
US20040102606A1 (en) 2001-04-24 2004-05-27 Danuta Balicki Histone H2A -derived peptides useful in gene delivery
US6805904B2 (en) 2002-02-20 2004-10-19 International Business Machines Corporation Process of forming a multilayer nanoparticle-containing thin film self-assembly
EP1499895B1 (en) * 2002-04-22 2008-05-07 University Of Florida Functionalized nanoparticles and methods of use
WO2004085998A2 (en) 2003-03-28 2004-10-07 The Children's Hospital Of Philadelphia Biomimetic hierarchies using functionalized nanoparticles as building blocks
US20050053668A1 (en) 2003-08-21 2005-03-10 Southwest Research Institute Skeletally targeted nanoparticles
EP1748796A4 (en) 2004-03-05 2008-08-27 Univ Texas MULTILAYER NANOMEDICINE DELIVERY SYSTEM AND METHOD
WO2005123142A1 (en) 2004-06-10 2005-12-29 Abraxis Bioscience, Inc. A method using inorganic nanoparticles as non-viral vectors for gene therapy
US20060088599A1 (en) 2004-08-02 2006-04-27 Prasad Paras N Amino functionalized ORMOSIL nanoparticles as delivery vehicles
WO2008103276A2 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
US20100196492A1 (en) 2007-03-08 2010-08-05 Green Jordan J Electrostatic coating of particles for drug delivery
US8389485B2 (en) 2007-10-29 2013-03-05 University Of Massachusetts Encapsulated nanoparticles for nucleic acid delivery
US8323618B2 (en) 2007-11-07 2012-12-04 University Of Houston System Ultrasmall superparamagnetic iron oxide nanoparticles and uses thereof
CN101970687A (zh) 2007-11-09 2011-02-09 东北大学 用于体内基因输送的自组装胶束样纳米颗粒
US7999025B2 (en) 2008-01-28 2011-08-16 University Of Utah Research Foundation Asymmetrically-functionalized nanoparticles organized on one-dimensional chains
US8324333B2 (en) 2008-06-05 2012-12-04 Wisconsin Alumni Research Foundation Anionic charge-dynamic polymers for release of cationic agents
CA2757831C (en) 2009-04-07 2017-09-26 Dow Agrosciences Llc Nanoparticle mediated delivery of sequence specific nucleases
PT2816112T (pt) * 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
WO2011096408A1 (ja) 2010-02-02 2011-08-11 国立大学法人 東京大学 複合体微粒子およびその製造方法、ならびに該複合体微粒子を用いた薬学組成物
US9074187B2 (en) 2011-03-21 2015-07-07 Board Of Trustees Of The University Of Arkansas Nanostructural materials that increase mineralization in bone cells and affect gene expression through miRNA regulation and applications of same
US20140115726A1 (en) * 2011-04-05 2014-04-24 Cellectis New tale-protein scaffolds and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
EP2863892B1 (en) 2012-06-20 2017-11-08 University Of Waterloo Mucoadhesive nanoparticle delivery system
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof

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