CN113710258A - Prpf31基因表达敲低提高体外分化的人细胞的存活 - Google Patents

Prpf31基因表达敲低提高体外分化的人细胞的存活 Download PDF

Info

Publication number
CN113710258A
CN113710258A CN202080030633.0A CN202080030633A CN113710258A CN 113710258 A CN113710258 A CN 113710258A CN 202080030633 A CN202080030633 A CN 202080030633A CN 113710258 A CN113710258 A CN 113710258A
Authority
CN
China
Prior art keywords
cells
nucleic acid
differentiated
agent
prpf31
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
CN202080030633.0A
Other languages
English (en)
Chinese (zh)
Inventor
查尔斯·E·默里
莎拉·杜普拉斯
约翰·拉玛基亚
詹姆斯·富盖特
威廉·罗伯·麦克莱伦
莉儿·帕本
斯科特·希斯
土田洋
斯蒂芬妮·A·塔克
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.)
University of Washington
Original Assignee
University of Washington
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 University of Washington filed Critical University of Washington
Publication of CN113710258A publication Critical patent/CN113710258A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61L—METHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L27/00—Materials for grafts or prostheses or for coating grafts or prostheses
    • A61L27/36—Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix
    • A61L27/38—Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix containing added animal cells
    • A61L27/3804—Materials for grafts or prostheses or for coating grafts or prostheses containing ingredients of undetermined constitution or reaction products thereof, e.g. transplant tissue, natural bone, extracellular matrix containing added animal cells characterised by specific cells or progenitors thereof, e.g. fibroblasts, connective tissue cells, kidney cells
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/65—MicroRNA
    • 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Reproductive Health (AREA)
  • Cardiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oral & Maxillofacial Surgery (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Transplantation (AREA)
  • General Engineering & Computer Science (AREA)
  • Dermatology (AREA)
  • Botany (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Rheumatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080030633.0A 2019-03-15 2020-03-13 Prpf31基因表达敲低提高体外分化的人细胞的存活 Pending CN113710258A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962818979P 2019-03-15 2019-03-15
US62/818,979 2019-03-15
PCT/US2020/022679 WO2020190739A1 (en) 2019-03-15 2020-03-13 Improved survival of human cells differentiated in vitro by prpf31 gene expression knockdown

Publications (1)

Publication Number Publication Date
CN113710258A true CN113710258A (zh) 2021-11-26

Family

ID=72520464

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080030633.0A Pending CN113710258A (zh) 2019-03-15 2020-03-13 Prpf31基因表达敲低提高体外分化的人细胞的存活

Country Status (7)

Country Link
US (1) US20220152278A1 (enExample)
EP (1) EP3937956A4 (enExample)
JP (1) JP2022525193A (enExample)
CN (1) CN113710258A (enExample)
AU (1) AU2020241251A1 (enExample)
CA (1) CA3129782A1 (enExample)
WO (1) WO2020190739A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4356931A4 (en) * 2021-06-16 2025-07-16 Chigenovo Co Ltd VARIANT OF PRPF31 AND ITS USE
US20250082689A1 (en) * 2021-07-21 2025-03-13 University Of Washington Compositions and methods for enhancing cardiomyocyte transplant engraftment
JP2025534556A (ja) * 2022-10-21 2025-10-16 バイオジェン・エムエイ・インコーポレイテッド 網膜色素変性症を治療するための組成物及び方法

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140328825A1 (en) * 2011-12-30 2014-11-06 Cellscript, Llc MAKING AND USING IN VITRO-SYNTHESIZED ssRNA FOR INTRODUCING INTO MAMMALIAN CELLS TO INDUCE A BIOLOGICAL OR BIOCHEMICAL EFFECT
WO2018078301A1 (fr) * 2016-10-28 2018-05-03 Université Pierre Et Marie Curie - Paris 6 (Upmc) Procédé de différenciation de cellules souches pluripotentes en cardiomyocytes

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
US7875451B2 (en) * 2006-01-19 2011-01-25 The University Of Washington Formulation to improve survival of transplanted cells
AU2008204541B2 (en) * 2007-01-09 2014-07-10 Lead Pharma Cel Models Ip B.V. Provision of new cardiomyocyte progenitor cells and cardiomyocytes derived therefrom
US9213999B2 (en) * 2007-06-15 2015-12-15 Kyoto University Providing iPSCs to a customer
AU2008346801A1 (en) * 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
EP2510087A2 (en) * 2009-12-11 2012-10-17 The Brigham and Women's Hospital, Inc. Pathogen restriction factors
PT3265568T (pt) * 2015-03-06 2020-08-20 Massachusetts Eye & Ear Infirmary Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31
JP7333271B2 (ja) * 2017-03-15 2023-08-24 ユニヴァーシティ オブ ワシントン 心筋細胞の成熟および生着を増強するための方法および組成物

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140328825A1 (en) * 2011-12-30 2014-11-06 Cellscript, Llc MAKING AND USING IN VITRO-SYNTHESIZED ssRNA FOR INTRODUCING INTO MAMMALIAN CELLS TO INDUCE A BIOLOGICAL OR BIOCHEMICAL EFFECT
WO2018078301A1 (fr) * 2016-10-28 2018-05-03 Université Pierre Et Marie Curie - Paris 6 (Upmc) Procédé de différenciation de cellules souches pluripotentes en cardiomyocytes

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LEILA AZIZZADEH PORMEHR等: "Human organotypic retinal flat-mount culture (HORFC) as a model for retinitis pigmentosa11", J CELL BIOCHEM, vol. 119, no. 8, pages 6775 - 6783 *

Also Published As

Publication number Publication date
EP3937956A1 (en) 2022-01-19
JP2022525193A (ja) 2022-05-11
US20220152278A1 (en) 2022-05-19
AU2020241251A1 (en) 2021-10-07
WO2020190739A1 (en) 2020-09-24
EP3937956A4 (en) 2022-12-28
CA3129782A1 (en) 2020-09-24

Similar Documents

Publication Publication Date Title
Li et al. Regulatory T-cells regulate neonatal heart regeneration by potentiating cardiomyocyte proliferation in a paracrine manner
JP2019201631A (ja) 治療用微小胞と幹細胞の合成
US8815593B2 (en) Induction of human embryonic stem cell derived cardiac pacemaker or chamber-type cardiomyocytes by manipulation of neuregulin signaling
CN102459576B (zh) 用于调节干细胞的组合物和方法及其应用
US20240368541A1 (en) Methods of generating mature corneal endothelial cells
US10443044B2 (en) Generating cardiac progenitor cells from pluripotent stem cells using isoxazole or isoxazole like compounds
US20250216377A1 (en) Compositions and methods for detecting cardiotoxicity
JP5921558B2 (ja) 心臓人工多能性幹細胞ならびに心筋の修復および再生に使用する方法
JP2011522520A (ja) 細胞の脱分化を行う方法
US20220152278A1 (en) Improved survival of human cells differentiated in vitro by prpf31 gene expression knockdown
US20180042969A1 (en) Compositions and methods for the reprogramming of cells into cardiomyocytes
US20130202649A1 (en) Activation of innate immunity for nuclear reprogramming of somatic cells
WO2011031875A2 (en) Cell-based compositions and uses thereof
US20220213444A1 (en) Compositions and methods for cellular reprogramming
WO2017177050A1 (en) Direct reprogramming of somatic cells into myogenic cells
US20240392252A1 (en) Generating cardiac progenitor cells from pluripotent stem cells using isoxazole or isoxazole like compounds
CN117500940A (zh) 原钙粘蛋白在诊断和治疗癌症的方法中的用途
US12173322B2 (en) Genome-edited induced pluripotent stem cells, and cells derived therefrom, and uses thereof
EP3941496A1 (en) Methods of promoting cellular maturation with ampk activators
US20240158788A1 (en) Methods and compositions used to modify chromatin architecture to regulate phenotype in aging and cancer
Sottile Mesenchymal stem cells generate distinc functional hybrids via cell fusion or entosis
WO2023070130A2 (en) Mirna reprogramming of smooth muscle cells into endothelial cells
HK40096970A (zh) 产生成熟角膜内皮细胞的方法
Buccini Cardiogenic differentiation of induced pluripotent stem cells for regeneration of the ischemic heart
Cassano et al. CT-0507 Accepted 10/21/2011 for publication in “Cell Transplantation”

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20211126

WD01 Invention patent application deemed withdrawn after publication