Accelerating Gene Editing Therapies for Rare, Neurological Conditions

In June, the National Institutes of Health’s National Institute of Neurological Disorders and Stroke made a five-year, $22.8 million grant to a group led by The Jackson Laboratory to develop gene-editing therapies for four rare, neurological conditions. The use of a platform approach to develop therapies for multiple indications follows other efforts on going at the National Institutes of Health in the area of gene therapies. We spoke to Steve Murray, associate professor at The Jackson Laboratory, about the promise of gene-editing, the work being done under the grant, and why the work could have broad implications for treating rare genetic neurological conditions.