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2018 Fiscal Year Final Research Report

Challenging of development of cancer cell specific treatment by means of somatic genome editing

Research Project

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Project/Area Number 16K15251
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Human pathology
Research InstitutionTohoku University (2017-2018)
Tokyo Women's Medical University (2016)

Principal Investigator

Furukawa Toru  東北大学, 医学系研究科, 教授 (30282122)

Project Period (FY) 2016-04-01 – 2019-03-31
Keywords膵臓癌 / ゲノム編集 / CRISPR-Cas9 / KRAS / アデノウィルス
Outline of Final Research Achievements

In this study, we tried to develop a novel cancer cell-specific therapeutic procedure by means of targeting cancer cell-specific mutations via CRISPR-Cas9 method. Mutation-specific CRISP-Cas9 was supposed to destruct activated oncogenes or replace defective tumor suppressor genes with functional normal genes. Most of pancreatic cancer cells harbor mutant KRAS genes in specific codons, so that we designed CRISPR-Cas9 vectors to target the mutated codons in the KRAS gene and found that they were effective for inhibiting proliferations of those cells in mutation specific manner.

Free Research Field

人体病理学

Academic Significance and Societal Importance of the Research Achievements

癌は3人に2人が罹患する疾患であり、依然として多くの場合不治であり、特に膵臓癌は予後が極めて不良で、5年生存率は10%未満であり、年間4万人近くが亡くなっている。膵臓癌は90%以上がKRASの機能亢進性変異を持ち、異常RASの機能を押さえなければ膵臓癌の根本的な治療にはならないことを示唆しているが、本研究成果はKRAS変異特異的ゲノム編集により膵臓癌細胞の増殖をコントロールできる可能性を示した。アデノウィルスで一過性体細胞性ゲノム編集を高効率に起こすことで臨床的な応用への可能性を拓き、これまでにない癌治療を実現できる可能性を示した。

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Published: 2020-03-30  

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