CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
Poojitha Pinjala, Kamatham Pushpa Tryphena, Renuka Prasad, Dharmendra Kumar Khatri, Woong Sun, Shashi Bala Singh, Dalapathi Gugulothu, Saurabh Srivastava, Lalitkumar K. Vora
Abstract
Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to correct the causative gene mutation and establish animal models in neurodegenerative disorders. Since no strategy developed until now could completely cure Parkinson's disease (PD), neuroscientists aspire to use gene editing technology, especially CRISPR/Cas9, to induce a permanent correction in genetic PD patients expressing mutated genes. Over the years, our understanding of stem cell biology has improved. Scientists have developed personalized cell therapy using CRISPR/Cas9 to edit embryonic and patient-derived stem cells ex-vivo. This review details the importance of CRISPR/Cas9-based stem cell therapy in Parkinson's disease in developing PD disease models and developing therapeutic strategies after elucidating the possible pathophysiological mechanisms.