The approach takes advantage of naturally occurring bacterial protein-RNA systems that use the RNA component to recognize viral DNA sequences and the enzyme Cas9 to cut the DNA. By adding new RNA sequences; specific DNA sequences can now be targeted for cutting. The result is a tool whose application could extend to new therapies for diseases such as Huntingtons disease, which appears to be caused by a single abnormal gene.
|Single striatal neuron (yellow) transfected with nuclear inclusion (orange) mHtt, other neurons in background (blue), from press release , Attribution 'Dr. Steven Finkbeiner, Gladstone Institute of Neurological Disease, The Taube-Koret Center for Huntington's Disease Research, and the University of California San Francisco'|