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Molecular Conjugates

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Gene Therapy and Gene Delivery Systems

Part of the book series: Advances in Biochemical Engineering/Biotechnology ((ABE,volume 99))

Abstract

Molecular conjugates are nanometer-sized entities consisting of synthetic materials (lipids, polycations, targeting agents, and so on) and nucleic acids. These composites are delivery vehicles that function to provide the transport of nucleic acids to sites of action. Recently, great progress has been made in the construction of these nonviral delivery vehicles and the understanding of how they function in cells and animals. Here, we review some of the important issues in assembling molecular conjugates and understanding their behavior in biological fluids, cells, and animals. One of the largest challenges in the field of molecular conjugates is how to integrate the components into a workable system that exploits the combined attributes of the components without suffering losses due to the assembly of the system. We discuss some of the difficulties involved in the assembly of a functioning delivery system for in vivo use.

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Abbreviations

ASGPR:

asialoglycoprotein receptor

bp:

base pairs

CD:

cyclodextrin

EGFR:

epidermal growth factor receptor

FR:

folate receptor

h:

hour(s)

IFN-γ:

interferon-γ

IL-6:

interleukin-6

IL-12:

interleukin-12

i.v.:

intravenous

min:

minute(s)

NAD(P)H:

dihydronicotinamide adenine dinucleotide (phosphate)

NF-κB:

nuclear factor-κB

NLS:

nuclear localization signal

nm:

nanometer(s)

PBS:

phosphate-buffered saline

pDNA:

plasmid DNA

PEG:

polyethylene glycol

PEI:

polyethylenimine

shRNA:

small hairpin RNA

siRNA:

small interfering RNA

Tf:

transferrin

TfR:

transferrin receptor

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Authors and Affiliations

  1. Chemical Engineering, 210-41, California Institute of Technology, 91125, Pasadena, CA, USA

    Jeremy Heidel, Swaroop Mishra & Mark E. Davis

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  1. Jeremy Heidel
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  2. Swaroop Mishra
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  3. Mark E. Davis
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Correspondence to Mark E. Davis .

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David V. Schaffer Weichang Zhou

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Heidel, J., Mishra, S., Davis, M.E. Molecular Conjugates. In: Schaffer, D.V., Zhou, W. (eds) Gene Therapy and Gene Delivery Systems. Advances in Biochemical Engineering/Biotechnology, vol 99. Springer, Berlin, Heidelberg. https://doi.org/10.1007/10_002

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