alexa Optimization of Non-Viral Gene Therapeutics Using Bilamellar Invaginated Vesicles
ISSN: 2157-7412

Journal of Genetic Syndromes & Gene Therapy
Open Access

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Review Article

Optimization of Non-Viral Gene Therapeutics Using Bilamellar Invaginated Vesicles

Nancy Smyth Templeton1*and Neil Senzer2

1Gradalis Inc., 2545 Golden Bear Drive, Suite 110, Carrollton, TX 75006

2Mary Crowley Cancer Research Centers, 1700 Pacific Avenue, Suite 1100, Dallas, TX 75201

*Corresponding Author:
Nancy S. Templeton
Director of Delivery Systems, Gradalis Inc.,
2545 Golden Bear Drive
Suite 110, Carrollton, TX 75006-2317, USA
Tel: 214-442-8130
E-mail: [email protected]

Received date: November 15, 2011; Accepted date:December 15, 2011; Published date: December 17, 2011

Citation: Templeton NS, Senzer N (2011) Optimization of Non-Viral Gene Therapeutics Using Bilamellar Invaginated Vesicles. J Genet Syndr Gene Ther S5:002. doi:10.4172/2157-7412.S5-002

Copyright: © 2011 Templeton NS, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

 

Abstract

Bilamellar invaginated vesicles (BIVs) are unique liposomal nanoparticles (NPs) that are highly efficient vehicles for intravenous (iv) delivery of encapsulated therapeutics including plasmid DNA. Systemic administration of therapeutics is required to effectively treat or cure metastatic cancer, certain cardiovascular diseases, and other acquired or inherited diseases. In addition to having extended half-life and stability in circulation, BIVs are nontoxic, nonimmunogenic, biodegradable and can be repeatedly administered without losing potency. Furthermore, BIVs encapsulating therapeutic agents can be modified to specifically enter the disease cells using small molecules that mimic beta turns incorporated on the surface of BIV complexes while focusing biodistribution by bypassing uptake
in non-target organs and tissues using reversible masking. These modifications do not alter the unique properties of the BIV delivery system that provide for its robust treatment of disease demonstrated in small and large animal models and in Phase I clinical trials. This review will cover the unique properties of BIVs, including its fusogenic entry into cells and its ability to penetrate tight barriers in vivo. Methods to further improve the overall delivery-expression system including further purification of plasmid DNA to eliminate colanic acid from all current commercially produced preparations, and enhanced or prolonged expression provided by plasmid design will also be discussed.

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