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MGMT-G156A-transduced autologous hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- Case Comprehensive Cancer Center
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
MGMT-G156A-transduced autologous hematopoietic stem cells is an experimental ex vivo gene therapy developed by the Case Comprehensive Cancer Center. The therapy involves the collection of a patient's own CD34+ hematopoietic stem and progenitor cells, which are then transduced with a retroviral vector carrying a mutant form of the O6-methylguanine-DNA methyltransferase (MGMT) gene containing the G156A mutation. This specific mutation renders the MGMT enzyme resistant to inhibition by O6-benzylguanine (O6-BG). The primary goal of this approach is myeloprotection; by reinfusing these modified cells into the patient, the bone marrow is shielded from the dose-limiting hematologic toxicity of alkylating chemotherapies (such as carmustine and temozolomide) when administered in combination with O6-BG. This allows for the use of intensified chemotherapy regimens to treat advanced solid tumors, non-Hodgkin's lymphoma, and gliomas.
- Other names
- therapeutic autologous lymphocytesmutant MGMT gene-modified CD34+ cellsMGMT-G156A-transduced CD34+ cells
02
Targets
AGT (O6-methylguanine-DNA methyltransferase)
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