Intrabody and Parkinson's disease.
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 (alpha-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 alpha-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.
Zhou
Chun
C
Department of Neurology, Columbia University, New York, NY 10032, USA.
Przedborski
Serge
S
D000818:Animals; D000906:Antibodies / Q000235:genetics / Q000276:immunology* / Q000627:therapeutic use*; D015316:Genetic Therapy; D006801:Humans; D010300:Parkinson Disease / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D015202:Protein Engineering; D051844:alpha-Synuclein / Q000235:genetics / Q000276:immunology
D000906:Antibodies; D051844:alpha-Synuclein