JP2016533322A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
silica coating
polymer bound
arginine
decrease
growth factor
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
JP2016516540A
Other languages
English (en)
Japanese (ja)
Other versions
JP6595459B2 (ja
JP2016533322A (ja
Filing date
Publication date
Application filed filed Critical
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
Anticipated expiration legal-status Critical

Links

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 (OSRAM) 2017-10-12
JP6595459B2 JP6595459B2 (ja) 2019-10-23

Family

ID=52689537

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 (OSRAM)
EP (2) EP3520822A1 (OSRAM)
JP (1) JP6595459B2 (OSRAM)
KR (1) KR20160060133A (OSRAM)
CN (1) CN105579068A (OSRAM)
AU (1) AU2014321215B2 (OSRAM)
CA (2) CA3175320A1 (OSRAM)
ES (1) ES2719112T3 (OSRAM)
IL (1) IL244585B (OSRAM)
PT (1) PT3049116T (OSRAM)
RU (1) RU2670512C2 (OSRAM)
WO (1) WO2015042585A1 (OSRAM)

Families Citing this family (62)

* 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
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
US10188749B2 (en) 2016-04-14 2019-01-29 Fred Hutchinson Cancer Research Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
JP7317706B2 (ja) 2016-12-14 2023-07-31 リガンダル インコーポレイテッド 核酸およびタンパク質ペイロード送達のための方法および組成物
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CA3049244A1 (en) 2017-01-05 2018-07-12 Fred Hutchinson Cancer Research Center Systems and methods to improve vaccine efficacy
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20210148918A1 (en) * 2018-04-17 2021-05-20 Purdue Research Foundation Modifying surface of a live cell and the uses thereof
EP3781683A4 (en) * 2018-04-18 2022-02-16 Ligandal, Inc. Methods and compositions for genome editing
EP3796894A4 (en) * 2018-04-24 2022-05-04 Ligandal, Inc. METHODS AND COMPOSITIONS FOR GENOMIC EDITING
EP3788094A2 (en) * 2018-04-27 2021-03-10 Genedit Inc. Cationic polymer and use for biomolecule delivery
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
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
US12415891B2 (en) 2018-09-25 2025-09-16 The University Of Tokyo Amphiphilic poly (amino acid), block copolyer using the amphiphilic poly (amino acid), and complex including the amphiphilic poly (amino acid) or the block copolymer and nucleic acid
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US12485136B2 (en) 2018-12-03 2025-12-02 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
CN113631709A (zh) 2018-12-20 2021-11-09 普拉克西斯精密药物股份有限公司 用于治疗kcnt1相关病症的组合物和方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021053246A1 (en) * 2019-09-20 2021-03-25 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
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
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)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
CA3162416C (en) 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
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)
WO2021231692A1 (en) 2020-05-15 2021-11-18 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)
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4341405A1 (en) 2021-05-20 2024-03-27 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
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
CA3234835A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
IL313660A (en) 2021-12-22 2024-08-01 Camp4 Therapeutics Corp Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs
AU2023283551A1 (en) 2022-06-10 2024-12-19 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
CN120435559A (zh) 2022-12-01 2025-08-05 4阵营疗法公司 使用靶向调节rna的反义寡核苷酸调控syngap1基因转录
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof

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
CN100419426C (zh) * 2002-04-22 2008-09-17 佛罗里达州立大学 功能化纳米微粒及其使用方法
WO2004085998A2 (en) 2003-03-28 2004-10-07 The Children's Hospital Of Philadelphia Biomimetic hierarchies using functionalized nanoparticles as building blocks
JP2007502833A (ja) 2003-08-21 2007-02-15 サウスウエスト・リサーチ・インスティチュート 骨格に標的とされるナノ粒子
EP1748796A4 (en) 2004-03-05 2008-08-27 Univ Texas METHOD AND SYSTEM FOR ADMINISTERING MULTILAYER NANOMEDICAMENTS
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
US20100015218A1 (en) 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
WO2008109806A2 (en) 2007-03-08 2008-09-12 Massachusetts Institute Of Technology Electrostatic coating of particles for drug delivery
JP2011517279A (ja) 2007-10-29 2011-06-02 ユニバーシティ オブ マサチューセッツ 核酸(siRNA)送達用の酵母細胞壁粒子(YCWP)多層状ナノ粒子
US8323618B2 (en) 2007-11-07 2012-12-04 University Of Houston System Ultrasmall superparamagnetic iron oxide nanoparticles and uses thereof
US20100285111A1 (en) 2007-11-09 2010-11-11 Northeastern University Self-assembling micelle-like nanoparticles for systemic gene delivery
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
TWI576432B (zh) 2009-04-07 2017-04-01 陶氏農業科學公司 序列特異性核酸酶之奈米粒子媒介式遞送技術
CA2783351C (en) * 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
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
EP2694091B1 (en) * 2011-04-05 2019-03-13 Cellectis Method for the generation of compact tale-nucleases and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
US20140005379A1 (en) 2012-06-20 2014-01-02 Frank GU Nanoparticle delivery system and components thereof
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

Similar Documents

Publication Publication Date Title
JP2016533322A5 (OSRAM)
JP2018100276A5 (OSRAM)
JP2018012698A5 (OSRAM)
HUS2300024I1 (hu) IX-es faktor polipeptid mutáns, alkalmazásai és eljárás elõállítására
JP2012500203A5 (OSRAM)
JP2010227108A5 (OSRAM)
JP2014132019A5 (OSRAM)
JP2015081257A5 (OSRAM)
CN105829298A (zh) 一种吡啶酮或嘧啶酮衍生物、及其制备方法和应用
JP2014098143A5 (OSRAM)
WO2017178653A3 (en) NEW ANTI-SIRPa ANTIBODIES AND THEIR THERAPEUTIC APPLICATIONS
EP3377656A4 (en) LOADING NUCLEIC ACIDS ON SUBSTRATES
EP3277806A4 (en) Methods of forming nanostructures using self-assembled nucleic acids, and nanostructures thereof
JP2017516475A5 (OSRAM)
EP3615085A4 (en) RNA NANOSTRUCTURES, THEIR MANUFACTURING PROCESSES AND THEIR USES
JP2019503884A5 (OSRAM)
JP2017501211A5 (OSRAM)
JP2016539641A5 (OSRAM)
EP3692155A4 (en) PRODUCTION OF POLYHYDROXYBUTYRATE IN WOOD-LJUNGDAHL MICRO-ORGANISMS
EP3868878A3 (en) Protein degradation inducing tag and usage thereof
JP2016031825A5 (OSRAM)
EP3904517A4 (en) OLIGOMERIC NUCLEIC ACID MOLECULE AND ITS USE
CN109563513A (zh) 一种抑制K-RAS基因表达的siRNA及其前体和应用
JP2016142759A5 (OSRAM)
JP2017528146A5 (OSRAM)