Study reveals significant restoration of retinal and visual function following Gene Therapy

(c) Nature Biomedical Engg

A breakthrough study, led by researchers from the University of California, Irvine, has demonstrated restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, “Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing,” illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

LCA patients gain benefit from Spark Therapeutics' gene therapy treatment, Luxturna

(c) Mass Eye & Ear

With the approval of Spark Therapeutics' Luxturna for Leber's Congenital Amaurosis (LCA), eye institutions around the US have started offering the treatment to their patients. In recent days, we have heard of this treatment being offered for the first time after the approval in Massachusetts Eye and Ear in Boston, followed the same day by the Children's Hospital of Los Angeles. A few days later, Bascom Palmer Eye Institute completed its first case.

FDA to soon decide approval for Spark Therapeutics' gene therapy for Leber Congenital Amaurosis

In the next few days, FDA panel will consider whether to advice approval for gene therapy for Leber's Congenital Amaurosis (LCA), which has been extensively tested in clinical trials by Spark Therapeutics.