RU2670512C2 - Доставка генов, опосредованная наночастицами, геномная коррекция и лиганд-направленная модификация в различных клеточных популяциях - Google Patents

Доставка генов, опосредованная наночастицами, геномная коррекция и лиганд-направленная модификация в различных клеточных популяциях Download PDF

Info

Publication number
RU2670512C2
RU2670512C2 RU2016115721A RU2016115721A RU2670512C2 RU 2670512 C2 RU2670512 C2 RU 2670512C2 RU 2016115721 A RU2016115721 A RU 2016115721A RU 2016115721 A RU2016115721 A RU 2016115721A RU 2670512 C2 RU2670512 C2 RU 2670512C2
Authority
RU
Russia
Prior art keywords
poly
cationic
nanoparticle
nanoparticles
polymer
Prior art date
Application number
RU2016115721A
Other languages
English (en)
Russian (ru)
Other versions
RU2016115721A3 (enExample
RU2016115721A (ru
Inventor
Шива Прасад КОТХА
Андре Роналд УОТСОН
Ваибхав А. ПАНДИТ
Original Assignee
Ренссилэйер Политекник Инститьют
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ренссилэйер Политекник Инститьют filed Critical Ренссилэйер Политекник Инститьют
Publication of RU2016115721A publication Critical patent/RU2016115721A/ru
Publication of RU2016115721A3 publication Critical patent/RU2016115721A3/ru
Application granted granted Critical
Publication of RU2670512C2 publication Critical patent/RU2670512C2/ru

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6923—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being an inorganic particle, e.g. ceramic particles, silica particles, ferrite or synsorb
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/95—Protection of vectors from inactivation by agents such as antibodies or enzymes, e.g. using polymers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nanotechnology (AREA)
  • Inorganic Chemistry (AREA)
  • Immunology (AREA)
  • Ceramic Engineering (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Polymers & Plastics (AREA)
RU2016115721A 2013-09-23 2014-09-23 Доставка генов, опосредованная наночастицами, геномная коррекция и лиганд-направленная модификация в различных клеточных популяциях RU2670512C2 (ru)

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
RU2016115721A RU2016115721A (ru) 2017-10-30
RU2016115721A3 RU2016115721A3 (enExample) 2018-03-21
RU2670512C2 true RU2670512C2 (ru) 2018-10-23

Family

ID=52689537

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2016115721A RU2670512C2 (ru) 2013-09-23 2014-09-23 Доставка генов, опосредованная наночастицами, геномная коррекция и лиганд-направленная модификация в различных клеточных популяциях

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2780664C2 (ru) * 2020-10-21 2022-09-28 федеральное государственное автономное образовательное учреждение высшего образования "Московский физико-технический институт (национальный исследовательский университет)" Способ получения частиц на основе гематита для доставки генетических конструкций в клетку

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050123600A1 (en) * 2001-01-02 2005-06-09 Trubetskoy Vladimir S. 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

Family Cites Families (25)

* 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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2295954C2 (ru) * 2000-09-28 2007-03-27 Чирон Корпорейшн Микрочастицы для доставки гетерологичных нуклеиновых кислот
US20050123600A1 (en) * 2001-01-02 2005-06-09 Trubetskoy Vladimir S. Compositions and methods for drug delivery using pH sensitive molecules

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
MIUATA K., GOUDA N. et al., Enhanced transfection with silica-coated polyplexes loading plasmid DNA // Biomaterials, 2010, 31, стр.4764-4770. *
MIUATA K., GOUDA N. et al., Enhanced transfection with silica-coated polyplexes loading plasmid DNA // Biomaterials, 2010, 31, стр.4764-4770. WANG C., LUO X. et al., Influence of the polyanion on the physic-chemical properties and biological activities of polyanion/DNA/polycation ternary polyplexes // Acta Biomaterialia, 2012, 8, стр.3014-3026. REILLY M.J., LARSEN J.D. et al., Histone H3 Tail Peptides and Poly(ethylenimine) Have Synergistic Effects for Gene Delivery // Molecular Pharmaceutics, 2012, 9, стр.1031-1040. *
REILLY M.J., LARSEN J.D. et al., Histone H3 Tail Peptides and Poly(ethylenimine) Have Synergistic Effects for Gene Delivery // Molecular Pharmaceutics, 2012, 9, стр.1031-1040. *
WANG C., LUO X. et al., Influence of the polyanion on the physic-chemical properties and biological activities of polyanion/DNA/polycation ternary polyplexes // Acta Biomaterialia, 2012, 8, стр.3014-3026. *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2821521C2 (ru) * 2018-12-28 2024-06-25 Эмрит Дженетикс Лтд Способы образования полиплексов
RU2780664C2 (ru) * 2020-10-21 2022-09-28 федеральное государственное автономное образовательное учреждение высшего образования "Московский физико-технический институт (национальный исследовательский университет)" Способ получения частиц на основе гематита для доставки генетических конструкций в клетку

Also Published As

Publication number Publication date
ES2719112T3 (es) 2019-07-08
US10526616B2 (en) 2020-01-07
EP3049116A4 (en) 2017-05-17
EP3520822A1 (en) 2019-08-07
CA2924535C (en) 2022-12-13
IL244585B (en) 2019-11-28
KR20160060133A (ko) 2016-05-27
IL244585A0 (en) 2016-04-21
AU2014321215A1 (en) 2016-04-21
RU2016115721A3 (enExample) 2018-03-21
PT3049116T (pt) 2019-04-03
AU2014321215B2 (en) 2020-07-16
CN105579068A (zh) 2016-05-11
EP3049116A1 (en) 2016-08-03
CA3175320A1 (en) 2015-03-26
EP3049116B1 (en) 2019-01-02
US20200181642A1 (en) 2020-06-11
JP2016533322A (ja) 2016-10-27
RU2016115721A (ru) 2017-10-30
JP6595459B2 (ja) 2019-10-23
WO2015042585A1 (en) 2015-03-26
CA2924535A1 (en) 2015-03-26
US20160230189A1 (en) 2016-08-11

Similar Documents

Publication Publication Date Title
AU2014321215B2 (en) Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations
Momin et al. The challenges and opportunities in the development of MicroRNA therapeutics: a multidisciplinary viewpoint
Pan et al. MicroRNA-activated hydrogel scaffold generated by 3D printing accelerates bone regeneration
Vaissière et al. A retro-inverso cell-penetrating peptide for siRNA delivery
Kim et al. RNA interference in vitro and in vivo using an arginine peptide/siRNA complex system
Cao et al. Linc02349 promotes osteogenesis of human umbilical cord‐derived stem cells by acting as a competing endogenous RNA for miR‐25‐3p and miR‐33b‐5p
Tagalakis et al. Multifunctional, self-assembling anionic peptide-lipid nanocomplexes for targeted siRNA delivery
Choi et al. Glutamine-chitosan modified calcium phosphate nanoparticles for efficient siRNA delivery and osteogenic differentiation
Reid et al. Canonical and non-canonical roles for ATG8 proteins in autophagy and beyond
Shi et al. Endocytic mechanisms and osteoinductive profile of hydroxyapatite nanoparticles in human umbilical cord Wharton’s jelly-derived mesenchymal stem cells
Selvadoss et al. Harnessing exosomes for advanced osteoarthritis therapy
Li et al. Highly efficient delivery of siRNA to a heart transplant model by a novel cell penetrating peptide-dsRNA binding domain
Gao et al. Targeting macrophage endocytosis via platelet membrane coating for advanced osteoimmunomodulation
Song et al. Design of an acid-activated antimicrobial peptide for tumor therapy
Liu et al. Delivery of miRNA‐29b using R9‐LK15, a novel cell‐penetrating peptide, promotes osteogenic differentiation of bone mesenchymal stem cells
Kawamura et al. Intracellular signal-responsive gene carrier for cell-specific gene expression
Lai et al. Immunomodulatory peptide DP7-C mediates macrophage-derived exosomal miR-21b to promote bone regeneration via the SOCS1/JAK2/STAT3 axis
Wang et al. Calcium-siRNA nanocomplexes optimized by bovine serum albumin coating can achieve convenient and efficient siRNA delivery for periodontitis therapy
Singh et al. Implications of siRNA therapy in bone health: silencing communicates
Wu et al. Creation of Artificial Subcellular Organelles Using Compartmentalized Escherichia coli Bodies for Artificial Metalloenzyme-Mediated Abiotic Catalysis in Eukaryotic Cells
WO2010151638A1 (en) Jnk inhibitors for use in treating spinal muscular atrophy
Gestin et al. Transfection of heat shock protein 70 kDa (HSP70)
Predescu et al. Impaired caveolae function and upregulation of alternative endocytic pathways induced by experimental modulation of intersectin‐1s expression in mouse lung endothelium
HK1225274A1 (en) Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations
HK1225274B (en) Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations