| 研究課題/領域番号 |
23K15315
|
|---|
| 研究種目 |
若手研究
|
| 配分区分 | 基金 |
|---|
| 審査区分 |
小区分54010:血液および腫瘍内科学関連
|
|---|
| 研究機関 | 筑波大学 |
|---|
研究代表者 |
|
|---|
| 研究期間 (年度) |
2023-04-01 – 2026-03-31
|
| 研究課題ステータス |
交付 (2023年度)
|
| 配分額 *注記 |
4,680千円 (直接経費: 3,600千円、間接経費: 1,080千円)
2025年度: 1,430千円 (直接経費: 1,100千円、間接経費: 330千円)
2024年度: 1,430千円 (直接経費: 1,100千円、間接経費: 330千円)
2023年度: 1,820千円 (直接経費: 1,400千円、間接経費: 420千円)
|
|---|
| キーワード | Gene therapy / Gene editing / Hematopoietic stem cell / Stem cell transplant / Cell therapy / Hematopoietic Stem Cells / Hematology / HSC Transplantation |
|---|
| 研究開始時の研究の概要 |
We aim to establish a novel treatment approach for hereditary hematopoietic diseases. We will employ hematopoietic stem cell expansion and gene editing to generate a corrected stem cell graft in a SCID model. The graft will be transplanted in utero, circumventing the need for toxic preconditioning.
|
| 研究実績の概要 |
In the past fiscal year, efforts were focused on establishing a method to robustly generate functional hematopoietic stem cells (HSC) from the peripheral blood of donor mice. Our protocol allows for 4 to 5-fold expansion of harvested peripheral blood-derived stem cells after about one week. These cells could be gene edited using the CRISPR/Cas9 system, generating enough cells for stable engraftment in a autologous transplant setting. Furthermore, we were able to publish our HSC gene editing system which enables the production of a large number of genetically modified, transplantable stem cells from a single cell clone. Our findings published in this report will greatly contribute to applying the intrauterine transplantation approach to the SCID model, as outlined in the research plan.
|
| 現在までの達成度 (区分) |
現在までの達成度 (区分)
2: おおむね順調に進展している
理由
Since the laboratory of the applicant has moved to a new institution, and due to subsequent short-term lack of appropriate equipment, the intrauterine transplantaion experiments could not be carried out as initlally planned. Furthermore, clinical responsibilities of the applicant has interfered with some time-consuming experiments.
|
| 今後の研究の推進方策 |
The new lab has since been set up and equipment for intrauterine transplantations should become available during May 2024.
|
