A New Treatment May Be Coming for Leber Congenital Amaurosis

Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes blindness.

The study was conducted on mice with a human form of the disease (specifically, LCA 2). These promising results could mean a new treatment option may be coming to Leber congenital amaurosis patients in the future.

For the mice that were treated, improvements lasted 120 days. For those untreated, no improvements were seen.

What’s even more exciting is that researchers think this discovery could benefit other forms of LCA besides LCA 2 which was the specific one they analyzed. Zheng-Rong Lu, who is a professor of Biomedical Engineering at the University and was the leader of this research, says that he believes the technology could be used to deliver practically any type of gene. That means the treatment could be beneficial for a multitude of populations.

You see, vision loss is a fairly common issue, even though Leber congenital amaurosis may not be.

As people age, it is not a rare thing to suffer some sort of vision impairment. This new technology may be able to help a variety of patients, not just those with LCA. They are currently looking at its potential for various different visual disorders, such as forms of macular degeneration.

Additionally, the treatment is so innovative because it isn’t expected to harm the liver or the kidneys, which has been a side effect of some previous treatment options for LCA. Researchers believe the treatment is so unique because it is so localized.

Read more about this discovery here, and keep an eye out for updates on its progression!

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