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

Biomaterials Research

Review

Renuka Prasad¹  Woong Sun¹  Dalapathi Gugulothu²  Saurabh Srivastava³  Shashi Bala Singh⁴  Poojitha Pinjala⁴  Kamatham Pushpa Tryphena⁴  Dharmendra Kumar Khatri⁴  Lalitkumar Vora⁵ 

[1] Department of Anatomy, Korea University College of Medicine, Moonsuk Medical Research Building, 73 Inchon-Ro, Seongbuk-Gu, 12841, Seoul, Republic of Korea;Department of Pharmaceutics, Delhi Pharmaceutical Sciences and Research University (DPSRU), 110017, New Delhi, India;Department of Pharmaceutics, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana-500037, Hyderabad, India;Molecular and Cellular Neuroscience Lab, Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana-500037, Hyderabad, India;School of Pharmacy, Queen’s University Belfast, 97 Lisburn Road, BT9 7BL, Belfast, UK;

关键词: Neurodegenerative disorder;    α-synuclein;    Gene editing;    Human pluripotent stem cells;    Embryonic stem cells;    Disease model;   

DOI  :  10.1186/s40824-023-00381-y

 received in 2023-03-17, accepted in 2023-04-16,  发布年份 2023

来源: Springer

【 摘 要 】

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

【 授权许可】

CC BY   
© The Author(s) 2023

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