AU2014321215B2 - Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations - Google Patents

Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations Download PDF

Info

Publication number
AU2014321215B2
AU2014321215B2 AU2014321215A AU2014321215A AU2014321215B2 AU 2014321215 B2 AU2014321215 B2 AU 2014321215B2 AU 2014321215 A AU2014321215 A AU 2014321215A AU 2014321215 A AU2014321215 A AU 2014321215A AU 2014321215 B2 AU2014321215 B2 AU 2014321215B2
Authority
AU
Australia
Prior art keywords
leu
arg arg
ala
val
gln
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.)
Active
Application number
AU2014321215A
Other languages
English (en)
Other versions
AU2014321215A1 (en
Inventor
Shiva Prasad KOTHA
Vaibhav A. PANDIT
Andre Ronald WATSON
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Rensselaer Polytechnic Institute
Original Assignee
Rensselaer Polytechnic Institute
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 Rensselaer Polytechnic Institute filed Critical Rensselaer Polytechnic Institute
Publication of AU2014321215A1 publication Critical patent/AU2014321215A1/en
Application granted granted Critical
Publication of AU2014321215B2 publication Critical patent/AU2014321215B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • 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)
  • Gastroenterology & Hepatology (AREA)
AU2014321215A 2013-09-23 2014-09-23 Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations Active AU2014321215B2 (en)

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 (2)

Publication Number Publication Date
AU2014321215A1 AU2014321215A1 (en) 2016-04-21
AU2014321215B2 true AU2014321215B2 (en) 2020-07-16

Family

ID=52689537

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2014321215A Active AU2014321215B2 (en) 2013-09-23 2014-09-23 Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations

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 (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
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
CA3033327A1 (en) 2016-08-09 2018-02-15 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
JP7588390B2 (ja) 2016-10-14 2024-11-22 プレジデント アンド フェローズ オブ ハーバード カレッジ 核酸塩基エディターのａａｖ送達
CA3045131A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CN110121336A (zh) 2017-01-05 2019-08-13 弗莱德哈钦森癌症研究中心 改善疫苗功效的系统和方法
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
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
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 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
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US20210148918A1 (en) * 2018-04-17 2021-05-20 Purdue Research Foundation Modifying surface of a live cell and the uses thereof
AU2019255725A1 (en) * 2018-04-18 2020-12-10 Ligandal, Inc. Methods and compositions for genome editing
EP3796894A4 (en) * 2018-04-24 2022-05-04 Ligandal, Inc. METHODS AND COMPOSITIONS FOR GENOMIC 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
TWI764022B (zh) * 2018-07-30 2022-05-11 中央研究院 凝聚複合物的治療性奈米粒子及其治療細菌的用途
JP2022502384A (ja) 2018-09-25 2022-01-11 国立大学法人 東京大学 両親媒性ポリアミノ酸、該両親媒性ポリアミノ酸を用いたブロックコポリマー、および該両親媒性ポリアミノ酸または該ブロックコポリマーと核酸とを含む複合体
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
AU2019406186A1 (en) 2018-12-20 2021-07-15 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of KCNT1 related disorders
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
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
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
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
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
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)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
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)
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)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
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
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
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
AU2022421244A1 (en) 2021-12-22 2024-07-11 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
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
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas

Citations (1)

* 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

Family Cites Families (26)

* 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
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
DE60320780D1 (de) 2002-04-22 2008-06-19 Univ Florida Funktionalisierte nanopartikel und verwendungsverfahren
WO2004085998A2 (en) 2003-03-28 2004-10-07 The Children's Hospital Of Philadelphia Biomimetic hierarchies using functionalized nanoparticles as building blocks
WO2005020965A2 (en) 2003-08-21 2005-03-10 Southwest Research Institute Skeletally targeted nanoparticles
US20070190155A1 (en) 2004-03-05 2007-08-16 Leary James F Molecular programming of nanoparticle systems for an ordered and controlled sequence of events for gene-drug delivery
WO2005123142A1 (en) 2004-06-10 2005-12-29 Abraxis Bioscience, Inc. A method using inorganic nanoparticles as non-viral vectors for gene therapy
WO2006017476A2 (en) 2004-08-02 2006-02-16 The Research Foundation Of State University Of New York Amino functionalized ormosil nanoparticles as delivery vehicles
CA2689042A1 (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
CA2704056A1 (en) 2007-10-29 2009-05-07 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
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
BRPI1015952B8 (pt) 2009-04-07 2022-06-28 Dow Agrosciences Llc Método de introdução de uma nuclease sequência-específica (ssn) em uma célula vegetal
NO2510096T3 (enExample) 2009-12-10 2015-03-21
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
KR101982360B1 (ko) * 2011-04-05 2019-05-24 셀렉티스 콤팩트 tale-뉴클레아제의 발생 방법 및 이의 용도
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
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof

Patent Citations (1)

* 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

Also Published As

Publication number Publication date
EP3049116B1 (en) 2019-01-02
EP3049116A1 (en) 2016-08-03
US10526616B2 (en) 2020-01-07
EP3520822A1 (en) 2019-08-07
AU2014321215A1 (en) 2016-04-21
RU2670512C2 (ru) 2018-10-23
RU2016115721A3 (enExample) 2018-03-21
CN105579068A (zh) 2016-05-11
CA2924535A1 (en) 2015-03-26
EP3049116A4 (en) 2017-05-17
RU2016115721A (ru) 2017-10-30
CA3175320A1 (en) 2015-03-26
JP6595459B2 (ja) 2019-10-23
CA2924535C (en) 2022-12-13
ES2719112T3 (es) 2019-07-08
US20200181642A1 (en) 2020-06-11
WO2015042585A1 (en) 2015-03-26
PT3049116T (pt) 2019-04-03
KR20160060133A (ko) 2016-05-27
US20160230189A1 (en) 2016-08-11
JP2016533322A (ja) 2016-10-27
IL244585A0 (en) 2016-04-21
IL244585B (en) 2019-11-28

Similar Documents

Publication Publication Date Title
AU2014321215B2 (en) Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations
US11713336B2 (en) Peptides and nanoparticles for intracellular delivery of molecules
Levingstone et al. Calcium phosphate nanoparticles-based systems for RNAi delivery: Applications in bone tissue regeneration
Suh et al. Peptide-mediated intracellular delivery of miRNA-29b for osteogenic stem cell differentiation
KR20240172763A (ko) 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
KR20210038841A (ko) 게놈 편집을 위한 방법 및 조성물
Liu et al. Delivery of miRNA‐29b using R9‐LK15, a novel cell‐penetrating peptide, promotes osteogenic differentiation of bone mesenchymal stem cells
Oh et al. Multimeric Amphipathic α‐Helical Sequences for Rapid and Efficient Intracellular Protein Transport at Nanomolar Concentrations
CN112584873A (zh) 用作细胞穿透肽的肽
Nirasawa et al. Development of A2G80 peptide-gene complex for targeted delivery to muscle cells
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
Ren et al. Self-Assembled Plasmid Delivery System for PPM1D Knockout to Reverse Tumor Malignancy
JP2022528325A (ja) 増強型トランスフェクション
Shen Understanding the Biological Barriers to Jurkat Transfection using Non-Viral Peptides
Pärnaste How, why, what and where: Mechanisms behind CPP/cargo nanocomplexes
Watanabe et al. Intracellular delivery of RNA via RNA-binding proteins or peptides
RU2637371C2 (ru) Гистоны и биодеградируемые липиды как средство для доставки нуклеиновых кислот в клетки эукариот
HK1245305B (en) Peptides and nanoparticles for intracellular delivery of molecules
HK1245305A1 (en) Peptides and nanoparticles for intracellular delivery of molecules

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)