Researchers must further validate these initial findings in larger animal models with eyes that more closely resemble human eyes. Because rodents experience a much more rapid progression of vision loss than do larger mammals, these experiments may take somewhat longer to gauge the treatments effectiveness. Optimal doses must also be established to insure that the gene or genetic information penetrates as many photoreceptor cells as possible. The safety of the gene delivery system must be tested to make sure it does not cause a harmful immune response. In science, gene delivery systems are called vectors.
Vectors act like a fleet of microscopic delivery trucks transporting genes into retinal cells.
Vectors are composed of genetically modified viruses. Viruses are extremely effective at infiltrating cells. Viral vectors are modified to remove their harmful qualities while still retaining their gene delivery capabilities. Although new-generation vectors are thought to be safe, Foundation researchers must establish their safety in the eye.