Powerful New Toolkit Targets Vision Loss in Advanced Retinal Degeneration

Powerful new toolkit targets vision loss in advanced retinal degeneration

Inherited retinal degenerations (IRDs) are a group of genetic disorders that lead to progressive vision loss as the light-sensing cells of the eye-the photoreceptors-die due to mutations in genes needed for their function and survival.

Novel gene therapy tools target inherited retinal degenerations at advanced stages

Inherited retinal degenerations (IRDs) are a group of genetic disorders that lead to progressive vision loss as the light-sensing cells of the eye—the photoreceptors—die due to mutations in genes needed for their function and survival.

Innovative Therapies Target Advanced-Stage Retinal Degenerations

In a groundbreaking advance poised to redefine therapeutic strategies for inherited retinal degenerations (IRDs), researchers at the University of Pennsylvania’s School of Veterinary Medicine have engineered a novel suite of photoreceptor-specific promoters designed to function effectively even during the advanced stages of retinal disease. This development confronts a persistent challenge in ocular gene therapy: delivering robust, targeted gene…