What Did the Research Discover?
Researchers studied retinitis pigmentosa (RP), an inherited eye disease that causes vision loss due to the death of light-sensitive cells in the retina. RP affects about 1 in 5,000 people worldwide, leading to symptoms like night blindness and tunnel vision. The condition is caused by genetic mutations, and more than 90 genes have been linked to its development.
To find better treatments, scientists are exploring gene therapy, a method that uses specially designed viruses or other tools to fix faulty genes. One of the most promising approaches is CRISPR-Cas9, a gene-editing technology that can correct disease-causing mutations in DNA. Researchers are also studying other methods, such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), which also modify genes to help slow or stop RP progression.
In recent studies, scientists have tested different gene-editing tools in lab-grown cells, animal models, and early-stage human trials. One treatment, called Luxturna (voretigene neparvovec), has already been approved for patients with mutations in the RPE65 gene. This gene therapy has helped improve vision in some people, though its long-term effects are still being studied.
How Can I Apply This Information?
This research is important because it shows that gene therapy may be a promising future treatment for RP. While no universal cure exists yet, scientists are working on ways to correct faulty genes and slow the disease’s progression. However, these treatments are still in the research phase, meaning they are being tested for safety and effectiveness before becoming widely available.
If you or someone you know has RP, it’s important to stay informed about new developments and talk to a healthcare provider about possible clinical trials. Gene therapy is not yet a standard treatment for most forms of RP, but ongoing studies suggest it could become a more common option in the future.
Source:
Liu Y, Zong X, Cao W, Zhang W, Zhang N, Yang N. Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress. Biomolecules. 2024 Jul 25;14(8):903.Available from:https://pubmed.ncbi.nlm.nih.gov/39199291/
