2023 Fiscal Year Research-status Report
Establishing an in utero gene therapy for genetic hematologic disorders
| Project/Area Number |
23K15315
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| Research Institution | University of Tsukuba |
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Principal Investigator |
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| Project Period (FY) |
2023-04-01 – 2026-03-31
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| Keywords | Gene therapy / Gene editing / Hematopoietic stem cell / Stem cell transplant |
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| Outline of Annual Research Achievements |
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.
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| Current Status of Research Progress |
Current Status of Research Progress
2: Research has progressed on the whole more than it was originally planned.
Reason
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.
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| Strategy for Future Research Activity |
The new lab has since been set up and equipment for intrauterine transplantations should become available during May 2024.
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