scholarly journals Targeting of Magnetic Nanoparticle-coated Microbubbles to the Vascular Wall Empowers Site-specific Lentiviral Gene Delivery in vivo

Theranostics ◽  
2017 ◽  
Vol 7 (2) ◽  
pp. 295-307 ◽  
Author(s):  
Yvonn Heun ◽  
Staffan Hildebrand ◽  
Alexandra Heidsieck ◽  
Bernhard Gleich ◽  
Martina Anton ◽  
...  
2012 ◽  
Vol 8 (8) ◽  
pp. 1309-1318 ◽  
Author(s):  
Hanna Mannell ◽  
Joachim Pircher ◽  
Franziska Fochler ◽  
Yvonn Stampnik ◽  
Thomas Räthel ◽  
...  

2016 ◽  
Vol 5 (21) ◽  
pp. 2799-2812 ◽  
Author(s):  
Yiyan He ◽  
Jie Zhou ◽  
Shengnan Ma ◽  
Yu Nie ◽  
Dong Yue ◽  
...  

2021 ◽  
Author(s):  
Hongyi Li ◽  
John E Heath ◽  
James S Trippett ◽  
Mikhail G. Shapiro ◽  
Jerzy O Szablowski

Targeted gene delivery to the brain is a critical tool for neuroscience research and has significant potential to treat human disease. However, the site-specific delivery of common gene vectors such as adeno-associated viruses (AAVs) is typically performed via invasive injections, limiting their scope of research and clinical applications. Alternatively, focused ultrasound blood-brain-barrier opening (FUS-BBBO), performed noninvasively, enables the site-specific entry of AAVs into the brain from systemic circulation. However, when used in conjunction with natural AAV serotypes, this approach has limited transduction efficiency, requires ultrasound parameters close to tissue damage limits, and results in undesirable transduction of peripheral organs. Here, we use high throughput in vivo selection to engineer new AAV vectors specifically designed for local neuronal transduction at the site of FUS-BBBO. The resulting vectors substantially enhance ultrasound-targeted gene delivery and neuronal tropism while reducing peripheral transduction, providing a more than ten-fold improvement in targeting specificity. In addition to enhancing the only known approach to noninvasively target gene delivery to specific brain regions, these results establish the ability of AAV vectors to be evolved for specific physical delivery mechanisms.


1998 ◽  
Vol 95 (20) ◽  
pp. 11886-11890 ◽  
Author(s):  
K. Ramani ◽  
Q. Hassan ◽  
B. Venkaiah ◽  
S. E. Hasnain ◽  
D. P. Sarkar

2006 ◽  
Vol 175 (4S) ◽  
pp. 323-324 ◽  
Author(s):  
Joseph Dall'era ◽  
Sweaty Koul ◽  
Jesse Mills ◽  
Jeremy Myers ◽  
Randall B. Meacham ◽  
...  

2000 ◽  
Author(s):  
Anne K. Kowal ◽  
Caroline Kohrer ◽  
Uttam L. RajBhandary

2020 ◽  
Vol 20 (11) ◽  
pp. 821-830
Author(s):  
Prasad Pofali ◽  
Adrita Mondal ◽  
Vaishali Londhe

Background: Current gene therapy vectors such as viral, non-viral, and bacterial vectors, which are used for cancer treatment, but there are certain safety concerns and stability issues of these conventional vectors. Exosomes are the vesicles of size 40-100 nm secreted from multivesicular bodies into the extracellular environment by most of the cell types in-vivo and in-vitro. As a natural nanocarrier, exosomes are immunologically inert, biocompatible, and can cross biological barriers like the blood-brain barrier, intestinal barrier, and placental barrier. Objective: This review focusses on the role of exosome as a carrier to efficiently deliver a gene for cancer treatment and diagnosis. The methods for loading of nucleic acids onto the exosomes, advantages of exosomes as a smart intercellular shuttle for gene delivery and therapeutic applications as a gene delivery vector for siRNA, miRNA and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and also the limitations of exosomes as a gene carrier are all reviewed in this article. Methods: Mostly, electroporation and chemical transfection are used to prepare gene loaded exosomes. Results: Exosome-mediated delivery is highly promising and advantageous in comparison to the current delivery methods for systemic gene therapy. Targeted exosomes, loaded with therapeutic nucleic acids, can efficiently promote the reduction of tumor proliferation without any adverse effects. Conclusion: In the near future, exosomes can become an efficient gene carrier for delivery and a biomarker for the diagnosis and treatment of cancer.


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