Delivering a Therapeutic Gene         Silencing a Disease Gene  

Delivering a Therapeutic Gene

The basic principle of gene therapy is to correct the genetic defect by putting the normal gene into the cells affected by the disease. The therapeutic gene needs to be delivered directly into the eye to the cells in which normal functioning of this gene is required. With the exception of the cornea, this requires surgery. The genes are delivered in a 'cassette' or 'vector' and research efforts have focused on designing the most appropriate vectors for the task. The currently favored delivery vectors are engineered viruses, which have demonstrated that it is possible to deliver therapeutic genes to the appropriate cells in the eye. Specific gene therapy strategies to replace genes which do not function have proved to be effective in a number of models of blindness, including a severe inherited retinal degeneration where recovery of visual function has been demonstrated. This significant progress is now being translated into clinical trials, and it is hoped that validation of these treatment strategies is just around the corner.
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