| Budget Amount *help |
¥17,030,000 (Direct Cost: ¥13,100,000、Indirect Cost: ¥3,930,000)
Fiscal Year 2023: ¥3,120,000 (Direct Cost: ¥2,400,000、Indirect Cost: ¥720,000)
Fiscal Year 2022: ¥3,510,000 (Direct Cost: ¥2,700,000、Indirect Cost: ¥810,000)
Fiscal Year 2021: ¥3,250,000 (Direct Cost: ¥2,500,000、Indirect Cost: ¥750,000)
Fiscal Year 2020: ¥3,510,000 (Direct Cost: ¥2,700,000、Indirect Cost: ¥810,000)
Fiscal Year 2019: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
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| Outline of Final Research Achievements |
We conducted gene therapy development research using stealth RNA vectors (SRV) for severe combined immunodeficiency. We have demonstrated that SRV can introduce genes into mouse hematopoietic stem cells/hematopoietic progenitor cells and CD34-positive cells derived from human umbilical cord blood efficiently. Differentiation of hematopoietic cells was confirmed by colony assay. When genes were introduced into common gamma chain-deficient cells using SRV, expression of the common gamma chain was achieved, and phosphorylation of STAT5 was confirmed upon stimulation with IL-2. Since it was sometimes difficult to maintain the proliferation of gene-transfected cells over a long period of time, we improved SRV and conducted a similar analysis. We will continue to improve SRV and apply it to other diseases.
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