Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders.
- Author:
Wooseok IM
1
;
Jangsup MOON
;
Manho KIM
Author Information
- Publication Type:Review
- Keywords: CRISPR/Cas9; gene editing; gene therapy; hereditary movement disorders
- MeSH: Biotechnology; Deoxyribonuclease I; DNA; Dystonia; Genetic Engineering; Genetic Therapy; Genome; Huntington Disease; Immune System; Movement Disorders*; Parkinson Disease; Spinocerebellar Degenerations; Streptococcus pyogenes
- From:Journal of Movement Disorders 2016;9(3):136-143
- CountryRepublic of Korea
- Language:English
- Abstract: Gene therapy is a potential therapeutic strategy for treating hereditary movement disorders, including hereditary ataxia, dystonia, Huntington's disease, and Parkinson's disease. Genome editing is a type of genetic engineering in which DNA is inserted, deleted or replaced in the genome using modified nucleases. Recently, clustered regularly interspaced short palindromic repeat/CRISPR associated protein 9 (CRISPR/Cas9) has been used as an essential tool in biotechnology. Cas9 is an RNA-guided DNA endonuclease enzyme that was originally associated with the adaptive immune system of Streptococcus pyogenes and is now being utilized as a genome editing tool to induce double strand breaks in DNA. CRISPR/Cas9 has advantages in terms of clinical applicability over other genome editing technologies such as zinc-finger nucleases and transcription activator-like effector nucleases because of easy in vivo delivery. Here, we review and discuss the applicability of CRISPR/Cas9 to preclinical studies or gene therapy in hereditary movement disorders.
