No. 3 Medical Innovation for 2018: Gene Therapy for Inherited Retinal Diseases (Video)
Gene therapy for inherited retinal diseases will be the No. 3 most influential medical innovation for 2018, predict experts at Cleveland Clinic.
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.
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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.