No. 3 Medical Innovation for 2018: Gene Therapy for Inherited Retinal Diseases (Video)

Finally a way to treat rare genetic vision disorders

In 2018, the FDA is anticipated to approve a new gene therapy for inherited retinal diseases. Delivering a new gene to targeted cells via viral “vectors” is expected to improve vision in some patients with Leber congenital amaurosis and retinitis pigmentosa.

Advertising Policy

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services Policy

Caused by biallelic RPE65 mutations, these rare genetic conditions cause progressive vision loss and blindness. Currently, there are no FDA-approved treatments. However, an innovative gene therapy delivers a new “normal” working copy of the gene that results in a functional protein. Researchers place this gene inside a modified virus, and this “vector” delivers it to retinal cells.

In this video, learn why gene therapy for inherited retinal diseases is No. 3 on Cleveland Clinic’s list of Top 10 Medical Innovations for 2018.