Intrabody and Parkinson's disease

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• 作者：Chun Zhou ; Serge Przedborski
• 关键词：Parkinson's disease ; Alpha-synuclein ; LRRK2 ; Intrabody ; scFv ; Gene-engineered antibody ; Molecular therapy ; Gene therapy
• 刊名：Biochimica et Biophysica Acta (BBA)/Molecular Basis of Disease
• 出版年：2009
• 出版时间：July 2009
• 年：2009
• 卷：1792
• 期：7
• 页码：634-642
• 全文大小：649 K

文摘

The intrabody technology has become a promising therapeutic avenue for a variety of incurable diseases. This technology is an intracellular application of gene-engineered antibodies, aimed at ablating the abnormal function of intracellular molecules. Parkinson's disease (PD) is a common neurodegenerative disease with no cure. Recent studies have explored possible intrabody applications against alpha-synuclein (α-syn), whose misfolding is believed to cause a familial form of PD. Here, we review the origin, production, and therapeutic mechanisms of intrabodies and the potential of intrabody protection against α-syn toxicity. Furthermore, we propose possible intrabody applications against leucine-rich repeat kinase 2 (LRRK2), whose mutations are the most frequent known cause of familial and sporadic PD.