Genome editing refers to a process of making precise and permanent changes in the genetic code of cells, tissues, and whole organisms. The first step in this process is to program an enzyme called a nuclease to bind to a precise DNA sequence, whereby the enzyme will cut the DNA. This double-stranded break will in turn induce the cell to make a repair at that site, which will change the DNA sequence. The repair mechanism can be utilized to either knock out or introduce selected genes. Given how rapidly gene editing systems are influencing diverse applications in biomedical research, biotechnology, and agriculture,1 clinical applications in neurology to repair genetic mutations causing disease, or to incorporate genes that might be of therapeutic benefit, are on the near horizon. The following cases illustrate how such an approach may be used to develop new ex vivo (case 1) and in vivo (case 2) therapies for neurologic disease, respectively, and highlight some of the translational challenges of using these molecular reagents safely and ethically in patients.

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