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Development of a photoreceptor-specific promoter for gene therapy

Jennie Marvelle 1, Donghyun Hong, PhD 1
1 Veterinary Pathobiology Department, Texas A&M College of Veterinary Medicine and Biomedical Sciences, College Station, Texas, USA

Abstract Jennie M Picture

Objective – To clone and characterize the human cone-rod homeobox promoter (CRX) and test its promoter activity in Y79 retinoblastoma cells.

Animals or Sample Population – Y79, HEK 293 cell lines

Procedure –We cloned 1.3 kb promoter region of human CRX  into the promoterless  reporter construct.  The promoter activity of this region was examined by transfection of promoter GFP reporter construct into human kidney and retinal cell lines.

Results – The cloned CRX promoter was able to drive expression of reporter in retinal cell lines, but not in kidney cell lines.    

Conclusions and Clinical Relevance –This study demonstrated that the CRX promoter region is sufficient to target expression of transgene to retinal cells and may be useful for gene therapy of photoreceptor degeneration.

Impact for Human Medicine – A photoreceptor-specific promoter  may enable gene therapy for humans and animals suffering from some forms of heritable retinal degeneration.