Department of Ophthalmology and Visual Sciences
Institute for Vision Research Center
Carver College of Medicine
Iowa City, Iowa

2020 BASIC RESEARCH PROJECT
Evaluating the Tropism and Transduction efficiency of Chimeric helper-dependent Adenoviral Vectors for Delivery of Large Genes

Scientific Summary: The two most common causes of inherited retinal degeneration are mutations found in two genes – ABCA4 [6.8 kilobases (kb) in length] and USH2A (15.6 kb).  Each of these genes are too large to fit into an adeno-associated virus (AAVs) which have a maximum packaging capacity of 4.7 kb, and are often used as vectors for delivering gene therapy.  As such, there remains a critical need for a large-capacity vector for gene therapy to treat blindness caused by mutations in genes that exceed the packaging limit of AAVs. The impact of the experiments proposed in this project will be the identification of a vector for the potential treatment of inherited blindness caused by genes too large for packaging into AAVs.

Dr. Wiley’s 2021 ARVO Poster Presentation