Fine-tuning gene delivery to combat childhood blindness

Researchers have used gene-carrying nanoparticles to prevent sight loss in mice with a human form of leber congenital amaurosis (LCA), in a study published in Molecular Therapy – Nucleic Acids. 

The inherited cause of blindness affects two to three babies in every 100,000 newborns, according to the National Institutes of Health. 

Although the research focused on leber congenital amaurosis 2, or LCA2, scientists believe the study holds promise for other forms of LCA and inherited diseases that lead to severe vision loss or blindness.

Study author Dr Zheng-Rong Lu, of Case Western Reserve University, told OT that using the nanoparticles for gene delivery had advantages over traditional viral forms of delivery. 

The nanoparticles were easy to produce, safe and had unlimited “cargo capacity,” Dr Lu explained. 

“Right now, genetic visual disorders are a major cause of retinal degeneration and severe vision loss, yet there are no approved therapies to treat these diseases,” Dr Lu shared.

“With gene therapy, we can cure the disease by delivering a healthy copy of the mutated gene directly to the cells that need to use it. However, this promise cannot be realized without a safe and effective gene delivery system to carry the gene into the target cells,” he emphasised. 

Gene replacement therapy using the delivery system in mice with LCA2 resulted in improved vision for more than 120 days. 

Dr Lu highlighted that while the gene delivery system had potential, further improvements were needed. Future work would focus on prolonging gene expression and improving tissue specificity.