Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?

genome editing tools, such as those based on CRISPR, have been increasingly utilized in both basic and translational neuroscience research. There are currently nine non-CNS genome editing therapies in clinical trials, and the pre-clinical pipeline of major biotechnology companies demonstrate that this number will continue to grow. Several biotechnology companies commercializing genome editing and modification technologies are developing therapies for CNS disorders with accompanying large partnering deals.