JP2023527766A - エキソソーム由来のＰＩＷＩ－ｉｎｔｅｒａｃｔｉｎｇ ＲＮＡおよびその使用方法 - Google Patents

エキソソーム由来のＰＩＷＩ－ｉｎｔｅｒａｃｔｉｎｇ ＲＮＡおよびその使用方法 Download PDF

Info

Publication number
JP2023527766A
JP2023527766A JP2022570643A JP2022570643A JP2023527766A JP 2023527766 A JP2023527766 A JP 2023527766A JP 2022570643 A JP2022570643 A JP 2022570643A JP 2022570643 A JP2022570643 A JP 2022570643A JP 2023527766 A JP2023527766 A JP 2023527766A
Authority
JP
Japan
Prior art keywords
pir
derived
pirna
exosome
seq
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.)
Pending
Application number
JP2022570643A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023527766A5 (https=
Inventor
マーバン，エドアルド
キウロ，アレッサンドラ
イブラヒム，アフマド，ジー．
Original Assignee
シーダーズ―シナイ メディカル センター
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 シーダーズ―シナイ メディカル センター filed Critical シーダーズ―シナイ メディカル センター
Publication of JP2023527766A publication Critical patent/JP2023527766A/ja
Publication of JP2023527766A5 publication Critical patent/JP2023527766A5/ja
Pending legal-status Critical Current

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/33—Fibroblasts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022570643A 2020-05-19 2021-05-17 エキソソーム由来のＰＩＷＩ－ｉｎｔｅｒａｃｔｉｎｇ ＲＮＡおよびその使用方法 Pending JP2023527766A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063027191P 2020-05-19 2020-05-19
US63/027,191 2020-05-19
PCT/US2021/070568 WO2021237238A1 (en) 2020-05-19 2021-05-17 Exosome-derived piwi-interacting rna and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2023527766A true JP2023527766A (ja) 2023-06-30
JP2023527766A5 JP2023527766A5 (https=) 2024-05-24

Family

ID=78707679

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022570643A Pending JP2023527766A (ja) 2020-05-19 2021-05-17 エキソソーム由来のＰＩＷＩ－ｉｎｔｅｒａｃｔｉｎｇ ＲＮＡおよびその使用方法

Country Status (6)

Country Link
US (1) US20230203487A1 (https=)
EP (1) EP4153743A4 (https=)
JP (1) JP2023527766A (https=)
CN (1) CN115698286A (https=)
AU (1) AU2021275327A1 (https=)
WO (1) WO2021237238A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3563859B1 (en) 2012-08-13 2021-10-13 Cedars-Sinai Medical Center Cardiosphere-derived exosomes for tissue regeneration
EP4494699A3 (en) 2014-10-03 2025-04-30 Cedars-Sinai Medical Center Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy
WO2017210652A1 (en) 2016-06-03 2017-12-07 Cedars-Sinai Medical Center Cdc-derived exosomes for treatment of ventricular tachyarrythmias
JP7336769B2 (ja) 2017-04-19 2023-09-01 シーダーズ―シナイ メディカル センター 骨格筋ジストロフィーを治療する方法及び組成物
WO2019126068A1 (en) 2017-12-20 2019-06-27 Cedars-Sinai Medical Center Engineered extracellular vesicles for enhanced tissue delivery
EP3749344A4 (en) 2018-02-05 2022-01-26 Cedars-Sinai Medical Center METHODS OF THERAPEUTIC USE OF EXOSOMES AND Y-RNAS
CN116875682B (zh) * 2023-07-08 2024-06-11 中国人民解放军总医院第二医学中心 用于诊断急性心肌梗死心脏损伤的piRNA标志物、试剂盒及其应用
WO2026035943A1 (en) 2024-08-08 2026-02-12 Cedars-Sinai Medical Center Formulations for oral delivery of nucleic acids
CN120485350B (zh) * 2025-04-12 2026-04-10 中国人民解放军总医院第二医学中心 用于诊断评估脓毒症心肌病的piRNA标志物、试剂盒及其应用
CN120713928A (zh) * 2025-06-30 2025-09-30 山西农业大学 miR-126及携带miR-126的外泌体在促进骨骼肌损伤修复和增殖分化中的应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3083997T (pt) * 2013-12-20 2020-11-03 Univ Lausanne Utilizações diagnósticas, prognósticas e terapêuticas de rnas longos não codificadores para doenças cardíacas e medicina regenerativa
CN104548136B (zh) * 2015-01-27 2018-02-13 青岛大学 一种piRNA药物组合物及其用途
WO2017147594A1 (en) * 2016-02-26 2017-08-31 Yale University COMPOSITIONS AND METHODS OF USING piRNAS IN CANCER DIAGNOSTICS AND THERAPEUTICS
HUE048369T2 (hu) * 2017-07-17 2020-07-28 Univ Masarykova Diagnosztikai eljárás végbélrák kimutatására
US11667916B2 (en) * 2017-09-08 2023-06-06 Korea University Research And Business Foundation Composition for preventing or treating liver fibrosis, containing exosome or exosome-derived ribonucleic acid
EP3749344A4 (en) * 2018-02-05 2022-01-26 Cedars-Sinai Medical Center METHODS OF THERAPEUTIC USE OF EXOSOMES AND Y-RNAS

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
CIRCULATION, vol. 136, no. 2, JPN6025024646, 2017, pages 200 - 214, ISSN: 0005625383 *
EUROPEAN HEART JOURNAL, vol. 38, JPN6025024650, 2016, pages 201 - 211, ISSN: 0005625387 *
EUROPEAN REVIEW FOR MEDICAL AND PHARMACOLOGICAL SCIENCES, vol. 22, no. 22, JPN6025024649, 2018, pages 7952 - 7961, ISSN: 0005625386 *
IRANIAN JOURNAL OF PHARMACEUTICAL RESEARCH, vol. 17, no. 1, JPN6025024648, 2018, pages 377 - 385, ISSN: 0005625385 *
JOURNAL OF CELLULAR PHYSIOLOGY, vol. 234, no. 12, JPN6025024647, 2019, pages 21732 - 21745, ISSN: 0005625384 *
STEM CELL REPORTS, vol. 2, no. 5, JPN6025024651, 2014, pages 606 - 619, ISSN: 0005625388 *
STEM CELLS AND DEVELOPMENT, vol. 29, no. 8, JPN6025024645, 15 April 2020 (2020-04-15), pages 511 - 520, ISSN: 0005625382 *

Also Published As

Publication number Publication date
US20230203487A1 (en) 2023-06-29
WO2021237238A1 (en) 2021-11-25
CN115698286A (zh) 2023-02-03
AU2021275327A1 (en) 2022-12-08
EP4153743A4 (en) 2025-09-24
EP4153743A1 (en) 2023-03-29

Similar Documents

Publication Publication Date Title
JP2023527766A (ja) エキソソーム由来のＰＩＷＩ－ｉｎｔｅｒａｃｔｉｎｇ ＲＮＡおよびその使用方法
US12584127B2 (en) Exosomes and micro-ribonucleic acids for tissue regeneration
US10767195B2 (en) Methods and products for expressing proteins in cells
ES3054848T3 (en) Stem cell-derived exosomes for the treatment of corneal scarring
CN101072866A (zh) 通过施用rna改变细胞性质的方法
US20210355492A1 (en) Cargo loaded extracellular vesicles
Oh et al. Lung regeneration therapy for chronic obstructive pulmonary disease
CA3157369A1 (en) Use of mbv for treating autoimmune disease
CN114574445B (zh) 永生化狗干细胞或其用途
US20240123034A1 (en) Mrnas encoding granulocyte-macrophage colony stimulating factor for treating parkinson's disease
JP2021522865A (ja) 栄養要求性調節可能な細胞を使用する遺伝子療法の方法および組成物
US20240374649A1 (en) Method for Treating Ilk Signaling Pathway Related Diseases Using Exosome Derived from Mesenchymal Stem Cells, and Pharmaceutical Composition
JP2022531930A (ja) 栄養要求性調節可能な細胞を使用する方法および組成物
Yuka et al. Current Approaches in Cardiac Repair: Somatic and Stem Cell Exosomes
Carbone et al. Placenta-Derived Stem Cells as a Source for Treatment of Lung and Liver Disease in Cystic Fibrosis
CN120037408A (zh) 药物组合物及其在制备治疗肝脏疾病的药物中的应用
AU2006207381A1 (en) Method of treatment by administration of RNA

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230124

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240516

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240516

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250624

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250924

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251119

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260210