Gene therapy and the regeneration of retinal ganglion cell axons.

Abstract

Because the adult mammalian central nervous system (CNS) has only limited intrinsic capacity to regenerate connections after injury, due to factors both intrinsic and extrinsic to the mature neuron (Shen et al., 1999; Berry et al., 2008; Lingor et al., 2008; Sun and He, 2010; Moore et al., 2011), therapies are required to support the survival of injured neurons and to promote the long-distance regrowth of axons back to their original target structures. The retina and optic nerve (ON) are part of the CNS and this system is much used in experiments designed to test new ways of promoting regeneration after injury (Harvey et al., 2006; Benowitz and Yin, 2008; Berry et al., 2008; Fischer and Leibinger, 2012). Testing of therapies designed to improve retinal ganglion cell (RGC) viability also has direct clinical relevance because there is loss of these centrally projecting neurons in many ophthalmic diseases.