Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders
Journal of Movement Disorders
;
: 136-143, 2016.
Artigo
em Inglês
| WPRIM
| ID: wpr-180368
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.
Texto completo:
DisponíveL
Índice:
WPRIM (Pacífico Ocidental)
Assunto principal:
Doença de Parkinson
/
Streptococcus pyogenes
/
Biotecnologia
/
DNA
/
Terapia Genética
/
Engenharia Genética
/
Degenerações Espinocerebelares
/
Genoma
/
Doença de Huntington
/
Desoxirribonuclease I
Idioma:
Inglês
Revista:
Journal of Movement Disorders
Ano de publicação:
2016
Tipo de documento:
Artigo
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