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Gene therapy fully restores vision in mouse model of Leber congenital amaurosis

Mice lacking the protein retGC1, which is deficient in humans suffering -1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success of this approach strongly support clinical testing of a targeted to the retinas of LCA1 patients, conclude the authors of the study published in Human , a peer-reviewed journal from , , publishers. The article is available free on the Human website until September 30, 2015.

Sanford Boye, Shannon Boye, and coauthors from , Gainesville, University of Oklahoma College of Medicine, Oklahoma City, and Salus University, Elkins Park, PA, emphasize the need for a treatment strategy targeting the loss of cone function that occurs in the eyes of patients with LCA1. They describe a gene replacement approach that uses an adeno-associated viral (AAV) vector to deliver the gene encoding the retGC1 protein to the cone-rich central retina in an all-cone mouse model deficient in retGC1. They report the study design, results, and their conclusions in the article “Gene Therapy Fully Restores Vision to the All-Cone Nrl-/-Gucy2e-/- Mouse Model of Leber Congenital Amaurosis-1.” DOI: 10.1089/hum.2015.053.

“This study shows the tremendous potential of recombinant (rAAV) gene therapy for the effective treatment of genetic causes of vision loss,” says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.