Development of upstream therapy targeting molecular basis of refractory cardiomyopathy
| Project/Area Number |
18K19543
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| Research Category |
Grant-in-Aid for Challenging Research (Exploratory)
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| Allocation Type | Multi-year Fund |
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| Review Section |
Medium-sized Section 53:Organ-based internal medicine and related fields
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| Research Institution | Osaka University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
肥後 修一朗 大阪大学, 医学系研究科, 寄附講座准教授 (00604034)
彦惣 俊吾 大阪大学, 医学系研究科, 准教授 (30423164)
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| Project Period (FY) |
2018-06-29 – 2020-03-31
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| Project Status |
Completed (Fiscal Year 2019)
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| Budget Amount *help |
¥6,240,000 (Direct Cost: ¥4,800,000、Indirect Cost: ¥1,440,000)
Fiscal Year 2019: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2018: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
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| Keywords | 心筋症 / ゲノム編集 / 疾患iPS細胞 / 難治性心筋症 |
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| Outline of Final Research Achievements |
Dilated cardiomyopathy and hypertrophic cardiomyopathy are the major underlying diseases of advanced heart failure requiring heart transplantation, and caused by genetic mutations. In this project, we conducted experimental research according to the following purpose; improvement of the efficiency of homology-directed repair in mouse cardiomyocytes, generation of human disease-specific induced pluripotent stem cells (iPSCs) from patients with cardiomyopathy with pathological genetic mutations, functional analysis using cardiomyocytes differentiated from iPSCs (iPSC-CMs) with precise allele modification via genome editing.
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| Academic Significance and Societal Importance of the Research Achievements |
指定難病のひとつである拡張型心筋症や肥大型心筋症は、遺伝子の変異により生じるとされています。本研究では、様々な分野に応用されているゲノム編集技術を、心筋症治療に応用することを検討しました。これまで、心臓の細胞である心筋細胞では、正確なゲノム編集である「相同組み換え」が生じないとされていました。本研究では、大人のマウスの心臓組織において相同組み換えが生じるか、またiPS細胞から作成したヒトの心筋細胞において相同組み換えが生じるかを検証しました。本研究から、心筋症研究におけるゲノム編集の更なる応用が期待されます。
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Report
(3 results)
Research Products
(24 results)
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[Journal Article] Long-term Effects of the Janus Kinase 1/2 Inhibitor Ruxolitinib on Pulmonary Hypertension and the Cardiac Function in a Patient with Myelofibrosis2020
Author(s)
Miyawaki H, Kioka H, Sato K, Kurashige M, Ozawa T, Shibayama H, Hikoso S, Morii E, Yamauchi-Takihara K, Sakata Y.
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Journal Title
Internal Medicine
Volume: 59
Issue: 2
Pages: 229-233
DOI
NAID
ISSN
0918-2918, 1349-7235
Year and Date
2020-01-15
Related Report
Peer Reviewed / Open Access
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[Presentation] High-content Image Analysis Combined with Genome Editing Clarifies the Role for Pkd1 as a Sarcoplasmic Reticulum Protein that Regulates Calcium Influx in Cardiomyocytes2019
Author(s)
Yuki Masumura, Shuichiro Higo, Takamaru Ishizu, Yasuaki Kohama, Mikio Shiba, Takumi Kondo, Satoshi Kameda, Hiroyuki Inoue, Tomoka Tabata, Satoki Nakamura, Seiji Takashima, Shigeru Miyagawa, Shungo Hikoso, Yasushi Sakata
Organizer
第3回BCVR
Related Report
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[Presentation] Rapid Evaluation of Candidate Causal Gene Mutation using High-content Image Analysis Combined with Targeted Gene Disruption2018
Author(s)
Yuki Masumura, Suzuka Kunimatsu, Shuichiro Higo, Yasuaki Kohama, Mikio Shiba, Takumi Kondo, Masato Shibamoto, Seiji Takashima, Shigeru Miyagawa, Shungo Hikoso, Yasushi Sakata
Organizer
AHA scientific sessions 2018（国際学会）
Related Report
Int'l Joint Research
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