JP2022525193A - Ｐｒｐｆ３１遺伝子発現ノックダウンによる、インビトロで分化したヒト細胞の生存の改善 - Google Patents

Ｐｒｐｆ３１遺伝子発現ノックダウンによる、インビトロで分化したヒト細胞の生存の改善 Download PDF

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JP2022525193A
JP2022525193A JP2021555433A JP2021555433A JP2022525193A JP 2022525193 A JP2022525193 A JP 2022525193A JP 2021555433 A JP2021555433 A JP 2021555433A JP 2021555433 A JP2021555433 A JP 2021555433A JP 2022525193 A JP2022525193 A JP 2022525193A
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チャールズ イー． マリー
サラ デュプラ
ジョン ラマッチア
ジェームス フガテ
ウィリアム ロブ マクレラン
リル パボン
スコット ティース
洋 土田
ステファニー エイ． タック
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ユニヴァーシティ オブ ワシントン
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Oral & Maxillofacial Surgery (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Transplantation (AREA)
  • General Engineering & Computer Science (AREA)
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  • 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)
JP2021555433A 2019-03-15 2020-03-13 Ｐｒｐｆ３１遺伝子発現ノックダウンによる、インビトロで分化したヒト細胞の生存の改善 Pending JP2022525193A (ja)

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

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JP2022525193A true JP2022525193A (ja) 2022-05-11
JP2022525193A5 JP2022525193A5 (enExample) 2023-03-17

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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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070166288A1 (en) * 2006-01-19 2007-07-19 University Of Washington Formulation to improve survival of transplanted cells
JP2011512326A (ja) * 2007-12-31 2011-04-21 ナノコア セラピューティクス，インコーポレイテッド 心不全の治療用のｒｎａ干渉
WO2011072247A2 (en) * 2009-12-11 2011-06-16 The Brigham And Women's Hospital, Inc. Pathogen restriction factors
JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
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
WO2018170280A1 (en) * 2017-03-15 2018-09-20 University Of Washington Methods and compositions for enhancing cardiomyocyte maturation and engraftment

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* 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
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
EP4721752A2 (en) * 2011-12-30 2026-04-08 Cellscript, Llc Herstellung und verwendung von in vitro-synthetisierter ssrna zur einführung in säugetierzellen zur induktion einer biologischen oder biochemischen wirkung

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070166288A1 (en) * 2006-01-19 2007-07-19 University Of Washington Formulation to improve survival of transplanted cells
JP2011512326A (ja) * 2007-12-31 2011-04-21 ナノコア セラピューティクス，インコーポレイテッド 心不全の治療用のｒｎａ干渉
WO2011072247A2 (en) * 2009-12-11 2011-06-16 The Brigham And Women's Hospital, Inc. Pathogen restriction factors
JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
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
WO2018170280A1 (en) * 2017-03-15 2018-09-20 University Of Washington Methods and compositions for enhancing cardiomyocyte maturation and engraftment

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PORMEHR, LEILA AZIZZADEH ET AL.: "Human organotypic retinal fl at-mount culture (HORFC) as a model for retinitispigmentosa11", JOURNAL OF CELLULAR BIOCHEMISTRY, vol. Volume 119, Issue 8, JPN6024002935, 10 May 2018 (2018-05-10), pages 6775 - 6783, ISSN: 0005247216 *

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EP3937956A1 (en) 2022-01-19
US20220152278A1 (en) 2022-05-19
AU2020241251A1 (en) 2021-10-07
CN113710258A (zh) 2021-11-26
WO2020190739A1 (en) 2020-09-24
EP3937956A4 (en) 2022-12-28
CA3129782A1 (en) 2020-09-24

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