CRISPR/Cas9 gene editing: A new hope for Alzheimer's disease

Alzheimer's disease (AD) is an increasingly prevalent and debilitating neurodegenerative condition characterized by cognitive impairment and the presence of amyloid beta (Ab) plaques and neurofibrillary tangles. It affects millions of individuals worldwide, and its incidence is rising rapidly. Central to our discussion is the recognition that AD's pathophysiology is closely intertwined with specific gene mutations, notably those involving presenilin (PSEN) and amyloid beta precursor protein (APP). Despite extensive clinical trials targeting these genetic factors, the desired efficiency has remained elusive. However, this obstacle can be overcome by an extraordinary genome editing tool called, clustered regularly interspaced short palindromic repeats (CRISPR/Cas9), which has poised to rectify aberrant genetic signatures associated with AD. This technology not only offers the prospect of amending troublesome gene mutations but has also ushered in the creation of valuable AD models, therapeutic avenues, and innovative diagnostic approaches. From in vitro experiments to in vivo models, CRISPR/Cas9 has significantly enriched the understanding of the intricacies of the nervous system, presenting exciting opportunities for advancements in the field of neurodegenerative disease research. Within this book chapter, we delve into the utilization of CRISPR/Cas9 gene editing as a potential tool in addressing AD and its associated clinical manifestations. While this tool has been widely utilized and known for treating various neurodegenerative diseases such as Parkinson's disease and Huntington's disease, our primary focus is on its application in AD. Along with this, we also highlight its status as an emerging technology with tremendous therapeutic potential.