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Allogeneic hematopoietic stem cell transplantation (Columbia University)
- Development stage
- Unknown
- Lead developer
- Columbia University
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Allogeneic hematopoietic stem cell transplantation (AHSCT) is a cellular therapy being investigated for the treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). MNGIE is a rare, fatal autosomal recessive disorder caused by mutations in the TYMP gene, which leads to a deficiency of the enzyme thymidine phosphorylase (TP). This deficiency results in systemic accumulation of thymidine and deoxyuridine, causing mitochondrial DNA instability and multi-organ failure. AHSCT aims to provide a permanent source of functional thymidine phosphorylase through the engraftment of donor hematopoietic cells, thereby normalizing nucleoside levels and halting disease progression. This specific research is led by Dr. Michio Hirano at Columbia University.
- Other names
- AHSCTAllogeneic bone marrow transplantationHematopoietic Allogeneic Stem Cells
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