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

Correction of neurological disease of Mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain-barrier gene delivery

2011 ◽  
Vol 102 (2) ◽  
pp. S18-S19
Author(s):  
Haiyan Fu ◽  
Julianne DiRosario ◽  
Smruti Killedar ◽  
Kimberly Zaraspe ◽  
Douglas McCarty
Keyword(s):  
Gene Delivery ◽  
Blood Brain Barrier ◽  
Neurological Disease ◽  
Brain Barrier ◽  
Adult Mice ◽  
Blood Brain

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 Endothelial Unc5B controls blood-brain barrier integrity

2021 ◽  
Author(s):  
Kevin Boyé ◽  
Luiz Henrique Medeiros Geraldo ◽  
Jessica furtado ◽  
Laurence Pibouin-fragner ◽  
Mathilde Poulet ◽  
...  
Keyword(s):  
Blood Brain Barrier ◽  
Brain Barrier ◽  
Proper Function ◽  
Downstream Signaling ◽  
Adult Mice ◽  
Blood Brain ◽  
The Central Nervous System ◽  
Specific Deletion ◽  
Blood Brain Barrier Integrity ◽  
Bbb Opening

Blood-brain barrier (BBB) integrity is critical for proper function of the central nervous system (CNS). Here, we showed that the endothelial Netrin1 receptor Unc5B controls BBB integrity by maintaining Wnt/β-catenin signaling. Inducible endothelial-specific deletion of Unc5B in adult mice led to region and size-selective BBB opening. Loss of Unc5B decreased BBB Wnt/β-catenin signaling, and β-catenin overexpression rescued Unc5B mutant BBB defects. Mechanistically, Netrin1 enhanced Unc5B interaction with the Wnt co-receptor LRP6, induced its phosphorylation and activated Wnt/β-catenin downstream signaling. Intravenous delivery of antibodies blocking Netrin1 binding to Unc5B caused a transient disruption of Wnt signaling and BBB breakdown, followed by neurovascular barrier resealing. These data identify Netrin-Unc5B signaling as a novel regulator of BBB integrity with potential therapeutic utility for CNS diseases.

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
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