Daniel Chung, DO, MA

University of Pennsylvania - Visionary

Project title: "In Utero Gene Therapy for Inherited Retinal Degenerations"

 

As many forms of retinal degeneration have an early onset, some at infancy and even before birth, the concept of early intervention gene therapy may have a beneficial impact. This study proposes to use cutting edge technology to delivery genes to mouse models of retinitis pigmentosa at an embryonic level, in hope of having greater ability in decrease, stop or reverse the progression of the disease. Although, the concept of human fetal gene therapy is of the distant future, the results of this study, may give better understanding and foundation for the earlier treatment of children with retinal degenerative disease with gene therapy at a neonatal level. 

This study will help us better understand the implications in intervening early in the  retinal development of inherited retinal degeneration retinas with gene therapy. It will give clues as to the best time to intervene, and if earlier intervention is better. It is anticipated that retinal recovery will be at least to the level of gene therapy conducted at later time-points and hope is for greater visual stability and recovery.