Gene Therapy to restore color blindness

A recent Nature letters paper by Mancuso et al details the use of a recombinant adeno-associated virus (rAAV) in gene delivery to restore the function of photo-pigments in squirrel monkeys. The male monkeys, red-green color-blind from birth, obtained the ability to process red-green color information via the delivery of the L-opsin gene into the retina. 

While this research opens the door for further hope of gene therapy involved in human eye conditions, including blindness, it also raises some interesting questions. It was previously thought that the brain of adult monkeys would be too "hard-wired" to gain a beneficial effect from the restoration of deficient pathways. The recent paper proves that gene therapy can be utilized in "middle aged" monkeys, and is not solely an avenue to be pursued during early development. This adds a lot of significance to work already begun in human patients, to restore an enzyme involved in a type of hereditary blindness.

Original Article:

Gene therapy for red-green colour blindness in adult primates

Katherine Mancuso, William W. Hauswirth, Qiuhong Li, Thomas B. Connor, James A. Kuchenbecker, Matthew C. Mauck, Jay Neitz & Maureen Neitz