Gene editing and central nervous system regeneration

Abstract Genome-editing technologies based on programmable nucleases have allowed the manipulation of several disease-causing genes, holding unprecedent potential to revolutionize the medical field. Among the currently available genome-editing technologies, the bacterial clustered regularly interspaced short palindromic repeat (CRISPR) system is the most rapidly developing class of endonucleases. Besides being affordable and well-suited for multiplexed gene-editing purposes, this widespread adoption is related to the simplicity with which this system can be designed and assembled. The ability to induce targeted deletions, insertions, and specific sequence alterations in living organisms is enabling reverse genetic studies of gene function, the generation of disease models, as well as the development of potential novel therapeutics for several human diseases. This chapter reviews the basic principles underlying the use of CRISPR-Cas9 as a genome-editing tool and its potential applications in the context of brain disorders, exploring key considerations and obstacles for clinical translation.