Dr. Lars Tebbe from University of Houston Awarded $90,000 Knights Templar Eye Foundation Grant for PRPH2 Mutation Research

Dr. Lars Tebbe from the University of Houston, Department of Biomedical Engineering located in Houston, Texas was awarded a $90,000 grant entitled: The clinical benefits of modulating ROM1 in switching a PRPH2-associated pattern dystrophy phenotype to retinitis pigmentosa.

Peripherin 2 (PRPH2) is important for the structural integrity of the light-sensitive retinal photoreceptor cells. Mutations in PRPH2 lead to different eye problems passed down in families, such as night blindness and or loss of color vision. Kids with mutations in PRPH2 can also develop serious eye conditions such as Leber Congenital Amaurosis (LCA) or Stargardt’s disease early in their childhood. The connection between PRPH2 and the pediatric eye diseases LCA and Stargardt’s disease underscores the relevance of PRPH2 as a target in the development of gene therapy for this vulnerable population. PRPH2 interacts with another protein called rod outer segment membrane protein 1 (ROM1). In a mouse model carrying a PRPH2 mutation that when ROM1 levels are reduced, it switches the diagnosis from daily vision loss to only a more manageable night blindness. This observation shows that ROM1 is a major player in reducing the severity of PRPH2 defects in daily vision. Dr. Tebbe aims to develop gene therapy strategies to help young patients with mutations in PRPH2. He will use special molecules called antisense oligonucleotides (ASOs) to reduce/eliminate ROM1. He will test whether administering the ASOs in eye drops will be as effective as injecting these ASOs formulated in nanoparticles inside the eye. This research should bring better treatments for kids with visual incapacitation due to mutations in PRPH2, clearly giving them a chance for a better life.