CRISPR technology for Parkinson’s disease: Recent advancements and ongoing challenges

Abstract

Parkinson’s disease (PD) is a neurodegenerative disorder caused by decreased dopamine, resulting in impaired motor function. Various gene editing methods are used in PD research to understand the disease’s complexity and develop treatments. With no cure and limited treatments, it is important to understand the recent advances in PD research, particularly with new gene editing technologies. Therefore, we evaluated recent advancements in gene therapy and CRISPR technology in PD research, using Pubmed to identify CRISPR use in PD research conducted within the past ten years. We compiled cell and gene therapy clinical trials for PD using clinicaltrials.gov, finding no current therapies approved for PD treatment, and CRISPR has yet to be incorporated in any clinical trials. We organized CRISPR technology used in PD research into three study types: animal models, stem cells, and cell culture. The studies reviewed involve research into genetic forms of PD and pathological hallmarks, such as α-synuclein accumulation, mitochondrial dysfunction, and cell death. Double or triple-transgenic models and induced pluripotent stem cells have been utilized more recently, contributing critical information to the understanding of PD. CRISPR is a powerful tool that has significantly advanced PD research. However, much research is still required to fully unravel the pathology and see whether CRISPR can be used in therapies to correct gene mutations and improve dysfunctional mechanisms across PD patients. Overall, CRISPR techniques for use in PD treatments are still in early development, being tested using cell and animal models that will hopefully move into clinical trials soon.