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Evaluation of tetrafunctional block copolymers as synthetic vectors for lung gene transfer

Paper ID Volume ID Publish Year Pages File Format Full-Text
5744 425 2015 8 PDF Available
Title
Evaluation of tetrafunctional block copolymers as synthetic vectors for lung gene transfer
Abstract

In the present study, we evaluated, in mice, the efficacy of the tetrafunctional block copolymer 704 as a nonviral gene delivery vector to the lungs. SPECT/CT molecular imaging of gene expression, biochemical assays, and immunohistochemistry were used. Our dataset shows that the formulation 704 resulted in higher levels of reporter gene expression than the GL67A formulation currently being used in a clinical trial in cystic fibrosis patients. The inflammatory response associated with this gene transfer was lower than that induced by the GL67A formulation, and the 704 formulation was amenable to repeated administrations. The cell types transfected by the 704 formulation were type I and type II pneumocytes, and transgene expression could not be detected in macrophages. These results emphasize the relevance of the 704 formulation as a nonviral gene delivery vector for lung gene therapy. Further studies will be required to validate this vector in larger animals, in which the lungs are more similar to human lungs.

Keywords
Tetrafunctional block copolymers; Lungs; Gene therapy; Nonviral vector; Molecular imaging
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Publisher
Database: Elsevier - ScienceDirect
Journal: Biomaterials - Volume 45, March 2015, Pages 10–17
Authors
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Subjects
Physical Sciences and Engineering Chemical Engineering Bioengineering
Get Full-Text Now
Don't Miss Today's Special Offer
Price was $35.95
You save - $31
Price after discount Only $4.95
100% Money Back Guarantee
Full-text PDF Download
Online Support
Any Questions? feel free to contact us