There is great interest in the development of gene therapy for inherited and degenerate disorders of the retina. However, in order to avoid adverse effects, it is important that the expression of the transgene is limited to its intended tissue. One way of achieving this is to regulate the transcription of the transgene using a cell-specific promoter, e.g., a promoter exclusively found in the cells of the cones or rods. Designing potency assays for gene therapy based on retina-specific promoters can be challenging.
Find out what these challenges are, and how they can be overcome.
