What did the research discover?
Scientists have been studying how changes in genes can lead to problems with vision. These changes affect two important parts of the eye: the retinal pigment epithelium and photoreceptors, which help us see. When these parts do not work correctly, they disrupt the way our eyes process images. Right now, there are no approved medications, gene therapies, or cell treatments for inherited eye diseases that cause blindness.
Researchers are exploring gene editing, which is a method of changing or fixing faulty genes. A new technology called CRISPR-Cas9 is at the forefront of these efforts. This tool allows scientists to cut and correct specific genes that cause eye diseases.
The study reviewed different ways to use gene editing:
- In laboratory settings (in vitro): Scientists tested CRISPR on induced pluripotent stem cells (iPSCs)—cells taken from patients that were modified to grow into eye cells. They successfully corrected genetic mutations in these cells. However, researchers worry about how well these corrected cells will function if transplanted back into a person’s eye.
- Inside the body (in vivo): Scientists experimented with injecting CRISPR-Cas9 directly into the eye to fix genetic mutations in living animals. Early studies in rodents (mice and rats) showed that this method could be safe and effective.
A clinical trial to test this approach in humans was planned for 2017. This was an important step toward seeing whether gene editing could be a real treatment for inherited eye diseases.
How can I apply this information?
This study shows that gene editing might one day be a treatment for inherited vision problems, but more research is needed. Scientists still have concerns about whether gene editing is completely safe and effective. There are two major challenges:
- Using gene editing in transplanted cells—Researchers must ensure these cells work properly when placed in the eye.
- Editing genes directly inside the eye—More testing is needed to confirm this method does not cause unintended harm.
Because this is still a developing area of research, gene editing is not yet available as a standard treatment. Scientists need to continue improving the technology before it can be used widely.
Source:
Smith AJ, Carter SP, Kennedy BN. Genome editing: the breakthrough technology for inherited retinal disease?. Expert Opinion on Biological Therapy. 2017 Oct 3;17(10):1245-54. https://pubmed.ncbi.nlm.nih.gov/28695744/
