CRISPR: a tool with potential for genomic reprogramming in neurological disorders

The intricate neural circuitry of the brain necessitates precise and synchronized transcriptional programs. Any disturbance during embryonic or adult development, whether caused by genetic or environmental factors, may result in refractory and recurrent neurological disorders. Inadequate knowledge of the pathogenic mechanisms underlying neurological disorders is the primary obstacle to the development of effective treatments, necessitating the development of alternative therapeutic approaches to identify rational molecular targets. Recently, with the evolution of CRISPR-Cas9 technology, an engineered RNA system provides precise and highly effective correction or silencing of disease-causing mutations by modulating expression and thereby avoiding the limitations of the RNA interference strategy. This article discusses the CRISPR-Cas9 technology, its mechanisms, and the limitations of the new technology. We provide a glimpse of how the far-reaching implications of CRISPR can open new avenues for the development of tools to combat neurological disorders, as well as a review of recent attempts by neuroscientists to launch therapeutic correction.

Automated summary

The complexity of neural circuitry requires precise transcriptional programs, and disturbance during development may lead to neurological disorders. Inadequate understanding of pathogenic mechanisms is a major obstacle, but the CRISPR-Cas9 technology offers a promising approach for targeted correction or silencing of disease-causing mutations. This article discusses the mechanisms, limitations, and potential applications of CRISPR technology in combating neurological disorders.