Glb 1 Gentransfer

Correction of a genetic defect in GM1-gangliosidosis in dog using zinc finger nucleases

The aim of the project is to develop a gene therapy approach for the repair of the ß-galactosidase mutation in Alaskan Huskey which causes GM1 gangliosidosis. For this approach zinc finger nucleases and other targeting vectors will be developed and tested in cell cultures and in an appropriate mouse models.

Coordinators: Prof. Dr. W. Baumgärtner, Ph.D., Prof. Dr. K. Schughart


Funding Agency

DFG - German Research Foundation