Retinal gene therapy 1998: summary of a workshop.

The 1998 Workshop on Retinal Gene Therapy evaluated the potential of gene therapy in treatment of retinal disease. Academic, industry, and private foundation representatives attended. Topics included: determing which retinal diseases are likely candidates for gene therapy, specific retinal degenerations and nonspecific neuronal survival mechanisms, design and use of viral and retroviral vectors in achieving regulated gene expression, animal models of retinal degeneration and associated therapies, human trials, and alternatives to gene therapy. The discussion of human trials explored the justification for moving from animal models to human testing, patient population concerns, lessons learned from previous human gene therapy trials, and the role of industry in support of basic and clinical research.