CRISPR/CAS9 GENOME EDITING FOR NEURODEGENERATIVE DISEASES

Nojadeh J. N., Eryilmaz N. S. B., ERGÜDER B. İ.

EXCLI JOURNAL, cilt.22, ss.567-582, 2023 (SCI-Expanded) identifier identifier

  • Yayın Türü: Makale / Derleme
  • Cilt numarası: 22
  • Basım Tarihi: 2023
  • Doi Numarası: 10.17179/excli2023-6155
  • Dergi Adı: EXCLI JOURNAL
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, EMBASE, Directory of Open Access Journals
  • Sayfa Sayıları: ss.567-582
  • Anahtar Kelimeler: Gene editing, neurodegenerative disorders, CRISPR, Cas9, Alzheimer's disease, Parkinson's disease, Huntington's disease, Amyotrophic lateral sclerosis, Spinocerebellar ataxia, PARKINSONS-DISEASE, GENE-THERAPY, EVOLUTIONARY CLASSIFICATION, ALPHA-SYNUCLEIN, CAS SYSTEMS, HUMAN-CELLS, IN-VIVO, MUTATION, IDENTIFICATION, POLYGLUTAMINE
  • Ankara Üniversitesi Adresli: Evet

Özet

Gene therapy has emerged as a promising therapeutic strategy for various conditions, including blood disorders, ocular disease, cancer, and nervous system disorders. The advent of gene editing techniques has facilitated the ability of researchers to specifically target and modify the eukaryotic cell genome, making it a valuable tool for gene therapy. This can be performed through either in vivo or ex vivo approaches. Gene editing tools, such as zinc finger nucleases, transcription activator-like effector nucleases, and CRISPR-Cas-associated nucleases, can be employed for gene therapy purposes. Among these tools, CRISPR-Cas-based gene editing stands out because of its ability to introduce heritable genome changes by designing short guide RNAs. This review aims to provide an overview of CRISPR-Cas technology and summarizes the latest research on the application of CRISPR/Cas9 genome editing technology for the treatment of the most prevalent neurodegenerative diseases including Alzheimer's disease, Parkinson's disease, Huntington's disease, Amyotrophic lateral sclerosis, and Spinocerebellar ataxia.