Development of mouse model and therapy of Diamond-Blackfan anemia

2017 Fiscal Year Final Research Report

• Project/Area Number: 26461596
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Nippon Medical School
• Principal Investigator: Miyake Koichi 日本医科大学, 医学部, 准教授 (90267211)
• Project Period (FY): 2014-04-01 – 2018-03-31
• Keywords: リボゾーム蛋白 / 貧血 / 先天異常 / siRNA / レンチウイルスベクター

Outline of Final Research Achievements

We generated tetracycline inducible lentiviral vector expressing siRNA against RPL5, RPL11, RPS24, and RPS17 to analyze the molecular mechanism of DBA (Diamond-Blackfan anemia). To develop DBA model mouse, first, we generated KRAB (Kruppel-associated box) gene transgenic mouse. Bone marrow cells from KRAB mouse were transduced with the above lentiviral vectors and induced with tetracycline. Although expression of KRAB was detected, it was difficult to get enough inducible expression after tetracycline induction. Now, we have a plan to analyze the reason and have another strategy to make a DBA model mouse using CRISPR-Cas9 system.

Free Research Field

血液内科学