Gene Editing Tool for Neurodegenerative Diseases

Neurodegenerative diseases (NDs) encompass a heterogeneous group of disorders characterized by the progressive degeneration and death of neural cells, as well as synaptic dysregulation in specific regions of the nervous system. These conditions, such as Alzheimer’s and Parkinson’s disease, are increasingly prevalent and have significant neurological implications, impacting millions of individuals worldwide. Various factors, including disrupted autophagy, signaling abnormalities, oxidative stress, mitochondrial dysfunction, impaired neurotrophins, and cytokines, contribute to the misfolding and aggregation of proteins, which are significant markers of NDs. Despite the growing prevalence of NDs, there is currently a lack of precise diagnostic tools and effective treatments for these conditions. However, recent advancements in genome sequencing and editing have opened up new possibilities for addressing these challenges. Emerging gene-editing tools (GETs), such as CRISPR-Cas9 and zinc finger nucleases (ZFNs), offer promising potential for correcting defective genes or DNA, leading to the restoration of lost neural function and connectivity. Furthermore, the integration of GETs with stem cell technology (SCT) holds tremendous prospects for advancing our comprehension of human genetics, regenerative medicine, and the biological mechanisms implicated in NDs. The present chapter aims to explore the main influential factors associated with NDs, providing a comprehensive overview of their evolutionary trajectory, methodology, molecular mechanisms, and applications of GETs. This knowledge serves as a foundation and forms the basis for developing preventive and targeted strategies against NDs. By leveraging the potential of gene editing, we can advance our understanding of the complexities surrounding NDs and improve the quality of life for affected individuals.