The study appears in the journal Molecular Therapy Nucleic Acids.The therapeutic effect lasted 120 days in treated mice.
The condition is one of the most common causes of blindness in children, according to the National Institutes of Health, affecting two to three of every 100,000 newborns.Leader Zheng-Rong Lu said that they believe this technology can deliver almost any type of gene to tackle inherited visual disorders.While other researchers focus on using modified viruses to deliver genes for therapy, sometimes the genes are too large for viruses to carry, Lu said. The protein is an essential constituent of the visual cycle that converts light to electrical signals to the brain.: A new gene-delivery system for an inherited form of blindness shows promise, according to a recent study.Researchers at Case Western Reserve University have developed gene-carrying nanoparticles that home in on target cells and prevent vision loss in mice with a human form of Leber congenital amaurosis.
They are also studying whether the nanoparticles can be used with the CRISPR/Cas9 gene-editing technique to treat genetic lesions related to retinal degenerative diseases.Though this research 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. The mutated gene fails to produce RPE65 protein in the retinal pigment epithelium (RPE), a cell layer critical for protecting photoreceptors (rods and cones). To track activity, Lus team included a fluorescent markerFollowing injection into the retina of mice,
The condition is one of the most common causes of blindness in children, according to the National Institutes of Health, affecting two to three of every 100,000 newborns.Leader Zheng-Rong Lu said that they believe this technology can deliver almost any type of gene to tackle inherited visual disorders.While other researchers focus on using modified viruses to deliver genes for therapy, sometimes the genes are too large for viruses to carry, Lu said. The protein is an essential constituent of the visual cycle that converts light to electrical signals to the brain.: A new gene-delivery system for an inherited form of blindness shows promise, according to a recent study.Researchers at Case Western Reserve University have developed gene-carrying nanoparticles that home in on target cells and prevent vision loss in mice with a human form of Leber congenital amaurosis.
They are also studying whether the nanoparticles can be used with the CRISPR/Cas9 gene-editing technique to treat genetic lesions related to retinal degenerative diseases.Though this research 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. The mutated gene fails to produce RPE65 protein in the retinal pigment epithelium (RPE), a cell layer critical for protecting photoreceptors (rods and cones). To track activity, Lus team included a fluorescent markerFollowing injection into the retina of mice,
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