Development of a new therapy for AML via CRISPR screen

2021 Fiscal Year Final Research Report

• Project/Area Number: 19K17859
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 54010:Hematology and medical oncology-related
• Research Institution: Kyushu University
• Principal Investigator: Yamauchi Takuji 九州大学, 大学病院, 助教 (20796213)
• Project Period (FY): 2019-04-01 – 2022-03-31
• Keywords: AML / novel therapy / CRISPR / HUSH / MORC3

Outline of Final Research Achievements

Whole genome CRISPR/Cas9 screen with ATRA was performed to develop a new treatment for AML. We identify HUSH component genes as synthetic lethal genes with ATRA for AML cells. Our results in this study indicated that the loss of MORC3 gene among the component of HUSH induced apoptosis with ATRA for AML cells.

Free Research Field

血液学

Academic Significance and Societal Importance of the Research Achievements

AMLは難治性の造血器悪性腫瘍であり、新たな治療法開発は喫緊の課題である。我々が用いた手法により、既存の抗白血病薬との新たな治療法開発が可能であると考える。今回の研究は、AML治療法における新たな治療法開発基盤となるシーズを創出した。