Genetic eye diseases usually result in a disruption of the retina and in many cases lead to considerable visual impairment and even blindness.
Researchers from the NMI Natural and Medical Sciences Institute in Reutlingen, the Faculty of Medicine at the University of Tübingen and Boehringer Ingelheim recently published results from a study to test carrier viruses for gene therapies in a human retina-on-chip system in "Stem Cell Reports". This novel system can be used to improve the development of future gene therapies for retinal diseases.
The specially produced therapeutic viruses are injected into the eye with a fine needle during treatment. However, the injected viruses do not cause the disease, but transport genetic material into the cells of the eye. The way they work is similar to the mRNA vaccines that became known as a result of the COVID-19 pandemic - the genetic material describes a blueprint that causes the cells at the injection site to produce a specific protein that is missing in the eye for the disease in question. In this way, the missing function of the protein can be restored in the patient.
Two treatment methods are currently available to transport the viruses to the diseased cells: intravitreal injection, in which the viruses are injected into the vitreous body of the eye, and subretinal injection, in which the viruses are injected directly under the outermost boundary layer of the retina. The development of new therapeutic viruses is lengthy and costly, not least because there is a lack of suitable non-clinical models that have predictive power for the human eye.
