CRISPR-Cas9 in Parkinson's Disease: A Potential Therapeutic Approach

CRISPR-Cas9 in Parkinson’s Disease: A Potential Therapeutic Approach Md.Ashrarul Hasiba*; Md. Riyadha and Sumiya Chowdhurya aDepartment of Pharmacy, Jagannath University, Dhaka, Bangladesh E-Mail- *Md.Ashrarul Hasib ([email protected]; +8801878359840) Md. Riyadh ([email protected]; +8801316213140) Sumiya Chowdhury ([email protected]; +8801859819427) Abstract: Parkinson’s disease (PD) is a progressive neurodegenerative disease characterized by the selective degeneration of dopaminergic neurons in the substantia nigra, resulting in motor and cognitive dysfunction. PD is one of the most common neurodegenerative diseases of the elderly and is a chronic progressive disorder characterized by loss of dopaminergic neurons in the substantia nigra and the development of a-triangle synonym, which cause different symptoms of PD including motor (tremors, rigidity, bradykinesia) and non-motor (mood disorders possible disorders gastrointestinal system), Despite the availability of symptomatic treatment (dopamine replacement therapy), no cure is available to stop or reverse the disease process. The CRISPR-Cas9 system for genome editing has recently developed as a powerful tool for precise manipulation of the genome, and can provide useful therapeutic strategies for PD. This study articulates the use of CRISPR-Cas9 tool in PD investigations and therapy, by correcting the mutation in genes, modulating gene expression and providing neuroprotection. Gene mutations such as SNCA, LRRK2, PINK1, and PRKN are associated with both familial and sporadic PD. CRISPR-Cas9 has been shown to correct pathogenic mutations in preclinical models that may restore normal cellular function. CRISPR-mediated transcriptional regulation also allows for the modulation of the expression of key neuroprotective genes (e.g., upregulation of dopamine synthesis, downregulation of α-synuclein aggregation, etc.). So, in recent years, several groups worked on introducing CRISPR components inside the body, including delivery through viral vectors or nanoparticle systems, to achieve high-efficiency and specific conditional targeting gene editing especially in neural tissues.CRISPR-Cas9 holds great promise, but also some potential pitfalls like off-target effects, ethical questions, and difficulties delivering it through the blood brain barrier that have made it with a large voter segment in the PD therapy marketplace. Refinements in base and prime editing, as well as improvements in non-viral delivery methods to improve safety and specificity of CRISPR-based therapies, are the subject of considerable interest. Whether CRISPR-Cas9 can be developed into an effective therapeutic approach to treat PD remains to be determined by continued research and clinical trials. This approach, if successful, may offer a long-term treatment solution to neurodegenerative disease, representing a serious leap forwards from hyper-targeted symptomatic therapies. Keywords: CRISPR-Cas9, Parkinson’s disease, gene therapy, neurodegeneration, genome editing.