However, there is a critical need for increased funding for the NEI to make these sightsaving treatments a reality. With increased support of the NEI, scientists can accelerate the pace of research and bring these promising treatments to the clinic. Without increased support, these treatments may very well never reach the clinic. Below is an overview of existing opportunities that with additional NEI funding could advance treatments and cures for patients with blinding retinal degenerative diseases. Gene Therapy Gene therapy is the introduction of a gene or genetic information into cells for the purpose of achieving a therapeutic effect.
Gene therapy can take different approaches based on the form of the disease.
For example, in autosomal recessive forms of retinal degenerative disease, both copies of the gene contain mutations. In recessive and some X-linked forms of retinal degeneration, the disease causing gene mutations often produce nonfunctional proteins. Lacking the protein, the retinal cells cannot function properly. In these cases, gene replacement therapy aims to deliver a healthy functioning gene directly to the affected cells so that the cell can function properly. In laboratory experiments, gene replacement therapy has already halted vision loss in animal models.