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

CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
  • Pinjala, Poojitha; 
  • Tryphena, Kamatham Pushpa; 
  • Prasad, Renuka; 
  • Khatri, Dharmendra Kumar; 
  • Sun, Woong; 
  • 외 4명
Citations

WEB OF SCIENCE

41
Citations

SCOPUS

51

초록

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.

키워드

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
제목
CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
제목 (타언어)
CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
저자
Pinjala, Poojitha; Tryphena, Kamatham Pushpa; Prasad, Renuka; Khatri, Dharmendra Kumar; Sun, Woong; Singh, Shashi Bala; Gugulothu, Dalapathi; Srivastava, Saurabh; Vora, Lalitkumar
DOI
10.1186/s40824-023-00381-y
발행일
2023-12-01
유형
Review
저널명
Biomaterials Research
권
27
호
1
페이지
1273 ~ 1290