Send to

Choose Destination
Gene Ther. 2005 Apr;12(8):639-46.

Gene therapy progress and prospects: targeted gene repair.

Author information

Department of Biological Sciences, Delaware Biotechnology Institute, University of Delaware, Newark, DE 19716, USA.


The capacity to correct a mutant gene within the context of the chromosome holds great promise as a therapy for inherited disorders but fulfilling this promise has proven to be challenging. However, steady progress is being made and the development of gene repair as a viable and robust approach is underway. Here, we present some of the recent advances that are helping to shape our thinking about the feasibility and the limitations of this technique. For the most part, these advances center on understanding the regulation of the reaction and validating its application in animal models.

[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Nature Publishing Group
Loading ...
Support Center