Developing a gene therapy for Stargardt disease
Professor Robert MacLaren has described his pioneering research in a Fight for Sight podcast episode
Stargardt is commonly caused by a fault in the ABCA4 gene.
Professor MacLaren explained that his research team has discovered a way of splitting the gene in two halves that overlap and recombine.
“In this research we were able to show that if we split the gene into two segments in a certain way, we could deliver it in two viral vectors,” he shared.
“Imagine it like building a bridge over a river and the bridge is too big to fit on one lorry so you have to use two lorries. The issue is working out where to cut the bridge to make sure when you put it back together it’s stable,” Professor MacLaren elaborated.
The research team hopes that the potential therapy will soon be tested in a clinical trial.
On the podcast, Professor MacLaren also discusses his research on retinitis pigmentosa and the future of robot-assisted eye surgery.
Image credit: Fight for Sight