Gene Therapy Safe and Effective Treatment for Common Form of Leber Congenital Amaurosis

September 20, 2011

The latest issue of the Archives of Ophthalmology reported on a study intended to determine the safety and efficacy of gene therapy to treat the form of Leber congenital amaurosis (LCA) caused by RPE65 gene mutations (RPE65 converts vitamin A into its usable form for vision).

The therapy involved subretinal injection into the worse functioning eye of recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene.

No systemic toxicity was detected, and visual function improved in all patients to different degrees, albeit improvements were localized to treated areas. Cone and rod sensitivities increased significantly in the study eyes but not in the control eyes.

The study demonstrated that it is possible to correct a defect in the retinal pigment epithelium (RPE) using gene transfer techniques.

Click here to read the abstract.




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