[A murine model of transitory optic neuropathy based on small interference RNA-induced OPA1 silencing in vivo (gene mutation associated with Kjer's disease)].

PURPOSE: Developing a murine model of OPA1 linked optic neuropathy.

METHODS

Intravitreal injections (in adult C57BL/6J mice) of small interference RNA (siRNA) specific to OPA1 were performed in the left eye. The right eye served as control, injected with nonspecific siRNA (siRNA scramble). Visual evoked potentials and flash electroretinograms were performed 5 and 12 days after injection. Three months after injection, microscopy of optic nerve sections was performed.

RESULTS

The electrophysiological tests showed a significant reduction in the VEP when the siRNA OPA1-injected eye was stimulated, compared with the control eye injected with siRNA scramble. The electroretinogram was normal in both eyes: no significant difference between the right and the left eye was found. Three months after injection, no measurable axonal degeneration was found in either eye.

CONCLUSION

The reduced expression of OPA1 based on RNA silencing in adult mice could induce reversible dysfunction of retinal ganglion cells.