The present invention relates to: an AAV-based PDE6B
viral vector for treating
retinitis pigmentosa, the AAV-based PDE6B
viral vector containing a tissue-specifically expressed PDE6A
promoter; and a use of thereof, and provides a
gene therapy for treating
retinitis pigmentosa caused by PDE6B
gene deficiency. The
in vivo therapeutic
efficacy of seven types of AAV5-PDE6B vectors was confirmed using an AAV by using a PDE6A
promoter that is tissue-specifically expressed in photoreceptor rod cells that develop
retinitis pigmentosa. An AAV5-PDE6A-450-PDE6B vector was selected as a candidate due to exhibiting strong tissue-specific expression in photoreceptor rod cells under even off-target conditions, unlike the
gene expression characteristics of an AAV5-CMV-PDE6B vector, and was tested so as to be
usable in the development of a gene therapeutic agent for treating PDE6B-deficient
retinitis pigmentosa patients. Therefore, the present invention, related to AAV5-PDE6B for
retinitis pigmentosa treatment and containing a tissue-specifically expressed PDE6A
promoter, provides
retinitis pigmentosa patients with an important treatment option having improved safety, and can be expected to have fundamental therapeutic effects compared to conventional treatments.