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UCL stem cell therapy (CellResearch Corporation)
- Development stage
- Preclinical
- Lead developer
- CellResearch
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
UCL stem cell therapy for Haemophilia A is a cell-based regenerative and gene therapy being developed by CellResearch Corporation. The therapy utilizes stem cells derived from the umbilical cord lining (UCL), which contains both epithelial and mesenchymal stem cell populations. These cells are genetically modified using site-specific insertion via electroporation to express the Factor VIII (FVIII) gene. The goal of the treatment is to provide a long-term, endogenous source of Factor VIII production to restore blood clotting ability in patients with Haemophilia A, a genetic disorder caused by a deficiency in this protein. Preclinical studies in haemophiliac mice have demonstrated that transplantation of these modified cells can maintain Factor VIII levels and restore normal clotting function.
- Other names
- Umbilical Cord Lining Stem Cell TherapyUCLSC therapyUCL stem cell therapy (Haemophilia A)
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