COL7A1-SIN retroviral vector

The COL7A1-SIN retroviral vector is a self-inactivating (SIN) retroviral vector designed for ex vivo gene therapy. Its primary purpose is to deliver a functional copy of the COL7A1 gene, which encodes type VII collagen, into patient cells. This gene therapy approach aims to correct the underlying genetic defect in Recessive Dystrophic Epidermolysis Bullosa (RDEB), a severe genetic skin disease caused by loss-of-function mutations in the COL7A1 gene. By introducing the healthy COL7A1 gene, the vector facilitates the production of functional type VII collagen, a crucial component for forming anchoring fibrils that ensure dermal-epidermal adherence. This restoration of collagen aims to prevent the characteristic skin blistering and associated complications of RDEB. The SIN design minimizes the risk of oncogenic events often associated with traditional retroviral vectors, and the expression of the COL7A1 gene is driven by human promoters, such as the elongation factor 1α (EF1α) or the COL7A1 promoter itself.

Other names

SIN COL7A1 retroviral vectorCOL7A1-encoding SIN retroviral vectorCOL-7A1-encoding SIN retroviral vectorCOL 7A1-encoding SIN retroviral vector