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

被引:18
作者
Pinjala, Poojitha [1 ]
Tryphena, Kamatham Pushpa [1 ]
Prasad, Renuka [2 ]
Khatri, Dharmendra Kumar [1 ]
Sun, Woong [2 ]
Singh, Shashi Bala [1 ]
Gugulothu, Dalapathi [3 ]
Srivastava, Saurabh [4 ]
Vora, Lalitkumar [5 ]
机构
[1] Natl Inst Pharmaceut Educ & Res NIPER Hyderabad, Dept Pharmacol & Toxicol, Mol & Cellular Neurosci Lab, Hyderabad 500037, Telangana, India
[2] Korea Univ, Coll Med, Dept Anat, Moonsuk Med Res Bldg,73 Inchon Ro, Seoul 12841, South Korea
[3] Delhi Pharmaceut Sci & Res Univ DPSRU, Dept Pharmaceut, New Delhi 110017, India
[4] Natl Inst Pharmaceut Educ & Res NIPER Hyderabad, Dept Pharmaceut, Hyderabad 500037, Telangana, India
[5] Queens Univ Belfast, Sch Pharm, 97 Lisburn Rd, Belfast BT9 7BL, North Ireland
关键词
Neurodegenerative disorder; alpha-synuclein; Gene editing; Human pluripotent stem cells; Embryonic stem cells; Disease model; GENE-THERAPY; DOPAMINERGIC-NEURONS; CRYSTAL-STRUCTURE; MOUSE MODEL; OPEN-LABEL; RAT MODEL; GAD GENE; PHASE-I; CAS9; COMPLEX;
D O I
10.1186/s40824-023-00381-y
中图分类号
R318 [生物医学工程];
学科分类号
0831 ;
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.
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页数:18
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