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COL7A1 gene-modified autologous fibroblasts
- Development stage
- Phase 1
- Lead developer
- King's College London
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intradermal
01
Overview
COL7A1 gene-modified autologous fibroblasts is an ex vivo gene therapy developed by King's College London in collaboration with Guy's and St Thomas' NHS Foundation Trust for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The therapy involves harvesting a patient's own fibroblasts and transducing them with a third-generation self-inactivating lentiviral vector containing a codon-optimized COL7A1 cDNA under the control of a human phosphoglycerate kinase (PGK) promoter. These modified cells are then injected intradermally to restore the production of type VII collagen (C7), which is the primary constituent of anchoring fibrils at the dermal-epidermal junction. This approach aims to correct the underlying genetic defect in RDEB, thereby improving skin integrity and reducing the severe blistering characteristic of the disease.
- Other names
- Lentiviral-mediated COL7A1 gene-modified autologous fibroblastsCOL7A1-transduced autologous fibroblastsCOL-7A1-transduced autologous fibroblastsCOL 7A1-transduced autologous fibroblasts
02
Targets
COL7A1 (Collagen type VII alpha 1 chain)
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