Gene therapy (GT) can be described as the in vivo transfer of DNA for therapeutic purposes. In the case of congenital retinal dystrophies (RD), GT can only be considered a successful treatment option if the gene transfer results in the restoration of vision at some level. Thus, a critical component of developing GT treatments for RDs is assessing the amount of functioning vision that is produced. In mouse models of RD, visual function after gene therapy is tested using techniques such as electroretinograms (ERGs) and retinoid analysis. A potentially useful addition to these tests would be a murine behavior-based technique, like those used with the RPE65 dog model, to demonstrate effective GT-induced visual recovery.
|Title of host publication||Retinal Degenerative Diseases|
|Editors||Joe G. Hollyfield, Robert E. Anderson, Matthew M. LaVail|
|Place of Publication||Boston|
|Number of pages||7|
|Publication status||Published - 2006|
|Name||Advances in Experimental Medicine and Biology|