Pluripotent Stem Cell Derived Neurons as In Vitro Models for Studying Autosomal Recessive Parkinson’s Disease (ARPD): PLA2G6 and Other Gene Loci

Gopurappilly, Renjitha

doi:10.1007/5584_2021_643

Cited by 3 publications

(1 citation statement)

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“…Endogenous α-syn is a substrate for CMA, and the posttranslational modification of α-syn has an inhibitory effect on this process [ 22 ]. Various animal and human studies have suggested the role of ROS in the pathogenesis of PD by intervening in the release of lysosomal proteases [ 24 ]. The proteasomal enzymes required for UPS were decreased in the SN region of PD patients.…”

Section: An Overview Of Parkinson's Diseasementioning

confidence: 99%

CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease

et al. 2023

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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. Graphical Abstract

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Section: An Overview Of Parkinson's Diseasementioning

confidence: 99%