scholarly journals Modifying layered double hydroxide nanoparticles with peptide to penetrate blood brain barrier for gene delivery

2017 ◽  
Author(s):  
Huali Zuo
Keyword(s):  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Layered Double Hydroxide ◽  
Brain Barrier ◽  
Blood Brain ◽  
Double Hydroxide ◽  
Layered Double Hydroxide Nanoparticles

Cellular uptake and gene delivery using layered double hydroxide nanoparticles

2013 ◽  
Vol 1 (1) ◽  
pp. 61-68 ◽  
Author(s):  
Shuangde Li ◽  
Jinghuan Li ◽  
Chengle J. Wang ◽  
Qiang Wang ◽  
M. Zameel Cader ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Layered Double Hydroxide ◽  
Cellular Uptake ◽  
Double Hydroxide ◽  
Layered Double Hydroxide Nanoparticles

Enhanced blood–brain barrier penetration and glioma therapy mediated by a new peptide modified gene delivery system

Biomaterials ◽  
2015 ◽  
Vol 37 ◽  
pp. 345-352 ◽  
Author(s):  
Hui Yao ◽  
Kaiyuan Wang ◽  
Yi Wang ◽  
Shanshan Wang ◽  
Jianfeng Li ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Delivery System ◽  
Brain Barrier ◽  
Gene Delivery System ◽  
Blood Brain ◽  
Barrier Penetration

scholarly journals Ultrasound-Mediated Blood-Brain Barrier Opening Improves Whole Brain Gene Delivery in Mice

Pharmaceutics ◽  
2021 ◽  
Vol 13 (8) ◽  
pp. 1245
Author(s):  
Marie-Solenne Felix ◽  
Emilie Borloz ◽  
Khaled Metwally ◽  
Ambre Dauba ◽  
Benoit Larrat ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Focused Ultrasound ◽  
Fold Increase ◽  
Brain Barrier ◽  
Whole Brain ◽  
External Threats ◽  
Blood Brain ◽  
The Brain

Gene therapy represents a powerful therapeutic tool to treat diseased tissues and provide a durable and effective correction. The central nervous system (CNS) is the target of many gene therapy protocols, but its high complexity makes it one of the most difficult organs to reach, in part due to the blood-brain barrier that protects it from external threats. Focused ultrasound (FUS) coupled with microbubbles appears as a technological breakthrough to deliver therapeutic agents into the CNS. While most studies focus on a specific targeted area of the brain, the present work proposes to permeabilize the entire brain for gene therapy in several pathologies. Our results show that, after i.v. administration and FUS sonication in a raster scan manner, a self-complementary AAV9-CMV-GFP vector strongly and safely infected the whole brain of mice. An increase in vector DNA (19.8 times), GFP mRNA (16.4 times), and GFP protein levels (17.4 times) was measured in whole brain extracts of FUS-treated GFP injected mice compared to non-FUS GFP injected mice. In addition to this increase in GFP levels, on average, a 7.3-fold increase of infected cells in the cortex, hippocampus, and striatum was observed. No side effects were detected in the brain of treated mice. The combining of FUS and AAV-based gene delivery represents a significant improvement in the treatment of neurological genetic diseases.

scholarly journals Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

2011 ◽  
Vol 19 (6) ◽  
pp. 1025-1033 ◽  
Author(s):  
Haiyan Fu ◽  
Julianne DiRosario ◽  
Smruti Killedar ◽  
Kimberly Zaraspe ◽  
Douglas M McCarty
Keyword(s):  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Neurological Disease ◽  
Brain Barrier ◽  
Adult Mice ◽  
Blood Brain

Blood–brain barrier abnormalities caused by exposure to HIV-1 gp120 — Protection by gene delivery of antioxidant enzymes

2010 ◽  
Vol 38 (2) ◽  
pp. 313-325 ◽  
Author(s):  
Jean-Pierre Louboutin ◽  
Beverly A.S. Reyes ◽  
Lokesh Agrawal ◽  
Christina R. Maxwell ◽  
Elisabeth J. Van Bockstaele ◽  
...  
Keyword(s):  
Antioxidant Enzymes ◽  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Brain Barrier ◽  
Blood Brain ◽  
Hiv 1

scholarly journals The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier

2011 ◽  
Vol 2011 ◽  
pp. 1-12 ◽  
Author(s):  
Ruben J. Boado ◽  
William M. Pardridge
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Trojan Horse ◽  
Brain Barrier ◽  
Viral Gene ◽  
Specific Gene ◽  
Gene Promoters ◽  
Blood Brain ◽  
The Brain

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.

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