Michigan State University gene therapy repairs damaged adult retinas

Researchers at Michigan State University have demonstrated that a single-dose gene therapy can physically repair damaged retinal connections in adult dogs. The study, published in ‘Molecular Therapy Advances’, focused on an inherited vision condition caused by mutations in the CaBP4 gene, which is essential for chemical signaling between retinal cells and the brain.

By injecting a harmless virus carrying a functional copy of the CaBP4 gene into the retinas of affected Whippet dogs, the team observed significant structural improvements. The treatment not only halted the progression of blindness but also prompted the expansion of the outer plexiform layer (OPL) and the growth of synaptic ribbons. These findings suggest that the adult mammalian retina possesses a level of plasticity and regenerative capacity previously thought impossible.

Veterinary ophthalmologist Billie Beckwith-Cohen noted that the therapy repaired existing abnormalities and added new structural components. This breakthrough challenges the long-held biological assumption that mature mammalian nerve cells cannot rebuild their architecture after significant degradation.