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TREATMENT FOR PARKINSONIAN PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
TREATMENT FOR PARKINSONIAN PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
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机译:LRRK2基因突变治疗帕金森病患者
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摘要
A method of treating Parkinson's Disease (PD) characterized by the presence of a mutant allele of leucine-rich repeat kinase 2 (LRRK2) gene in a subject is disclosed. The method comprises administering to the subject a CRISPR-Cas system guide RNA (gRNA) which specifically binds to the mutant allele of said leucine-rich repeat kinase 2 (LRRK2) gene and a CRISPR endonuclease, thereby treating the Parkinson's Disease (PD).
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