Intrabody and Parkinson's disease

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

卷:1792

Review

Zhou, Chun¹  Przedborski, Serge^1,2,3 

[1] Columbia Univ, Dept Neurol, New York, NY 10032 USA

[2] Columbia Univ, Dept Pathol & Cell Biol, New York, NY 10032 USA

[3] Columbia Univ, Ctr Motor Neuron Biol & Dis, New York, NY 10032 USA

关键词: Parkinson's disease;    Alpha-synuclein;    LRRK2;    Intrabody;    scFv;    Gene-engineered antibody;    Molecular therapy;    Gene therapy;   

DOI  :  10.1016/j.bbadis.2008.09.001

来源: Elsevier

【 摘 要 】

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. (C) 2008 Elsevier B.V. All rights reserved.

【 授权许可】

Free