Infection with adeno-associated virus may protect against excitotoxicity

GENE therapy has developed as a promising approach for therapy in a broad v ariety of conditions. Viral vectors have been developed that may replace a defective gene, prevent expression of a mutant gene, or deliver a protectiv e gene and thereby delay cellular loss. Using adeno-associated virus contai ning green fluorescent protein (AAV-GFP) we were able to specifically trans duce cells located in the inner retina and induce over-expression of GFP in adult rat retinae. The delivery and expression of GFP had no influence the mselves:on retinal ganglion cell survival. Administration of the reporter v ector AAV-GFP provided retinal ganglion cells with slight but significant p rotection from intravitreal NMDA. This was a locally mediated phenomenon; g reater protection was seen in regions:with more transduced cells. Any evalu ation of the efficacy of a putative viral vector should consider the possib le protective or toxic effect of the native virus. (C) 1999 Lippincott Will iams & Wilkins.