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JMD : Journal of Movement Disorders

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Letter to the editor
A Case of AOA2 With Compound Heterozygous SETX Mutations
Hee Jin Chang, Ryul Kim, Minchae Kim, Jangsup Moon, Man Jin Kim, Han-Joon Kim
J Mov Disord. 2022;15(2):178-180.   Published online December 24, 2021
DOI: https://doi.org/10.14802/jmd.21139
  • 1,235 View
  • 206 Download
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Review Article
Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders
Wooseok Im, Jangsup Moon, Manho Kim
J Mov Disord. 2016;9(3):136-143.   Published online September 21, 2016
DOI: https://doi.org/10.14802/jmd.16029
  • 18,249 View
  • 608 Download
  • 8 Citations
AbstractAbstract PDF
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.

JMD : Journal of Movement Disorders