Blindness cured by targeted gene therapy

Braille by Zesmerelda

Last week, researchers published “the first example of cone-targeted gene therapy” in the advance online publication of Nature Medicine, in which they cured mice of a form of blindness.

Researchers from the University of Florida and the Jackson Laboratory in Maine were able to target and deliver corrective genes specifically to cone cells in young mice with achromatopsia and restore vision within two months. (Achromatopsia is a genetic condition causing cone vision loss in one in 30,000 in the US and leads to permanent central vision loss, extreme light sensitivity and colour blindness.)

The team, led by Dr. Bo Chang, created a harmless adeno-associated virus construct with cone cell specificity and outfitted it with corrective genes. The construct particles were then injected into the subretinal space of mouse eyes. At two months, electroretinogram tests and visual acuity tests confirmed that vision was fully restored both functionally and behaviorally in the affected mice.

This targeted therapy opens up the possibility of treating all major vision disorders that can lead to blindness including cone-rod dystrophies, late-stage retinitis pigmentosa, age-related macular degeneration and diabetic retinopathy.

With all the efforts in stem cells and gene therapy, blindness will be a thing of the past. Helen Keller would be proud!

Read more about this research in the New York Times: “Gene Therapy Used to Cure Mice Blindness.”