The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier
Joint Authors
Pardridge, William M.
Boado, Ruben J.
Source
Issue
Vol. 2011, Issue 2011 (31 Dec. 2011), pp.1-12, 12 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2011-11-16
Country of Publication
Egypt
No. of Pages
12
Main Subjects
Abstract EN
The application of blood-borne gene therapy protocols to the brain is limited by the presence of the blood-brain barrier (BBB).
Viruses have been extensively used as gene delivery systems.
However, their efficacy in brain is limited by the lack of transport across the BBB following intravenous (IV) administration.
Recent progress in the “Trojan Horse Liposome” (THL) technology applied to transvascular non-viral gene therapy of the brain presents a promising solution to the trans-vascular brain gene delivery problem.
THLs are comprised of immunoliposomes carrying nonviral gene expression plasmids.
The tissue target specificity of the THL is provided by peptidomimetic monoclonal antibody (MAb) component of the THL, which binds to specific endogenous receptors located on both the BBB and on brain cellular membranes, for example, insulin receptor and transferrin receptor.
These MAbs mediate (a) receptor-mediated transcytosis of the THL complex through the BBB, (b) endocytosis into brain cells and (c) transport to the brain cell nuclear compartment.
The expression of the transgene in brain may be restricted using tissue/cell specific gene promoters.
This manuscript presents an overview on the THL transport technology applied to brain disorders, including lysosomal storage disorders and Parkinson's disease.
American Psychological Association (APA)
Boado, Ruben J.& Pardridge, William M.. 2011. The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier. Journal of Drug Delivery،Vol. 2011, no. 2011, pp.1-12.
https://search.emarefa.net/detail/BIM-461296
Modern Language Association (MLA)
Boado, Ruben J.& Pardridge, William M.. The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier. Journal of Drug Delivery No. 2011 (2011), pp.1-12.
https://search.emarefa.net/detail/BIM-461296
American Medical Association (AMA)
Boado, Ruben J.& Pardridge, William M.. The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier. Journal of Drug Delivery. 2011. Vol. 2011, no. 2011, pp.1-12.
https://search.emarefa.net/detail/BIM-461296
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-461296