Lead by talented Dr. Kyle Fink, working in collaboration with Dr. David Segal, Department of Genetics at UC Davis, graduate student Peter Deng and other students in our group are working on a highly exciting Gene Editing project for Juvenile Huntington's disease (JHD).
They are developing gene editing strategies using TALENs. These are molecules that go into the neural progenitor cells and actually cut out the extra CAG repeats, reducing them to the normal range of 15. So those cells should be "fixed" and go on to make neurons in the brain that are not affected by HD. This is what we need for the JHD kids- repaired neurons to be made for the rest of their- hopefully long- lives. TALES can also block expression from specific genes such as the mutant htt gene and this work is also in progress.