Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders.
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.
Department of Neurology, Neuroscience Research Center, Seoul National University Hospital, Seoul, Korea.
Wooseok
W
Im
Department of Neurology, Neuroscience Research Center, Seoul National University Hospital, Seoul, Korea.
Jangsup
J
Moon
Department of Neurology, Neuroscience Research Center, Seoul National University Hospital, Seoul, Korea.|Protein Metabolism Medical Research Center, Seoul National University College of Medicine, Seoul, Korea.
Manho
M
Kim
CRISPR/Cas9; gene editing; gene therapy; hereditary movement disorders
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