Gene delivery is a process which helps to induce Deoxyribonucleic acid (DNA) into the host cells to decode the protein product and treat genetic disorders. Very recently there has been a profound discovery in gene delivery to prevent vision loss. Researchers have developed gene carrying nanoparticles that home in on target cells and prevent vision loss in animal models of an inherited eye disease, which is also one of the most common causes of blindness in children.
This research was published in the journal Molecular Therapy and it stated, “Nucleic acids focused on the form of the disease called Leber congenital amaurosis 2, or LCA2, the scientists and engineers involved in the study believe the technology holds promise for other forms of LCA as well as other inherited diseases that lead to severe vision loss or blindness’.
Zheng-Rong Lu, a lead research and Professor at Case Western Reserve University in Cleveland, Ohio, US was quoted saying that “We believe this technology can deliver almost any type of gene to tackle inherited visual disorders,”
Those with LAC2 carry a mutated RPE65 gene and suffer from profound vision loss from birth. The mutated gene fails to produce RPE65 protein in the retinal pigment epithelium (RPE), a cell layer critical for protecting photoreceptors (rods and cones).
The protein is an essential constituent of the visual cycle that converts light to electrical signals to the brain. Lu and colleagues designed a lipid-based nanoparticle called ECO to deliver healthy RPE65 genes to RPE cells.
“The promise of this technology is it localises the drug to the photoreceptor cells, sparing the liver and kidney from exposure,” Krzysztof Palczewski from the Case Western Reserve School of Medicine said.
A study was conducted in which the retina of the mice was injected, the researchers could see fluorescent green concentrating in RPE cells. Testing showed a significant increase in light-induced electrical activity from the eyes to the brain, indicating the rods and cones were operating as they should in the visual cycle. Conclusions made at the end of the test were that the therapeutic effect lasted 120 days in treated mice and there were no improvements observed in the untreated mice.
Palczewski remarked, “This work is important beyond one disease. The loss of photoreceptor cells affects virtually all of us.”