CRISPR/Cas9-mediated genome-editing of long-term hematopoietic stem cells

• Project/Area Number: 21K15872
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 52050:Embryonic medicine and pediatrics-related
• Research Institution: Jichi Medical University
• Principal Investigator: スブド ビャンバー 自治医科大学, 医学部, 助教 (90834193)
• Project Period (FY): 2021-04-01 – 2023-03-31
• Project Status: Granted (Fiscal Year 2021)
• Budget Amount: ¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000); Fiscal Year 2022: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000); Fiscal Year 2021: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
• Keywords: CRISPR/Cas9 / Hematopoietic stem cell / SCID / NHEJ / HDR

Outline of Research at the Start

In the proposed study, I will develop HDR-edited long-term HSC for autologous transplantation to cure X-SCID in the mice. In the future, this approach can be easily converted to the genome-editing of human true HSC and translate to the large animal study.