Abstract
Delivering therapeutic cargos to specific cell types in vivo poses many technical challenges. There is currently a plethora of drug leads and therapies against numerous diseases, ranging from small molecule compounds to nucleic acids to peptides to proteins with varying binding or enzymatic functions. Many of these candidate therapies have documented potential for mitigating or reversing disease symptoms, if only a means for gaining access to the intracellular target were available. Recent advances in our understanding of the biology of cellular uptake and transport processes and the mode of action of bacterial protein toxins have accelerated the development of toxin-based cargo-delivery vehicle platforms. This review provides an updated survey of the status of available platforms for targeted delivery of therapeutic cargos, outlining various strategies that have been used to deliver different types of cargo into cells. Particular emphasis is placed on the application of toxin-based approaches, examining critical issues that have hampered realization of post-intoxication antitoxins against botulism.
Keywords: Antitoxin, botulinum neurotoxin, neuromuscular junction, paralysis, passive immunization, post-exposure therapeutics, single-chain antibodies, synaptic function, tetanus neurotoxin.
Current Topics in Medicinal Chemistry
Title:Cargo-Delivery Platforms for Targeted Delivery of Inhibitor Cargos Against Botulism
Volume: 14 Issue: 18
Author(s): Brenda A. Wilson and Mengfei Ho
Affiliation:
Keywords: Antitoxin, botulinum neurotoxin, neuromuscular junction, paralysis, passive immunization, post-exposure therapeutics, single-chain antibodies, synaptic function, tetanus neurotoxin.
Abstract: Delivering therapeutic cargos to specific cell types in vivo poses many technical challenges. There is currently a plethora of drug leads and therapies against numerous diseases, ranging from small molecule compounds to nucleic acids to peptides to proteins with varying binding or enzymatic functions. Many of these candidate therapies have documented potential for mitigating or reversing disease symptoms, if only a means for gaining access to the intracellular target were available. Recent advances in our understanding of the biology of cellular uptake and transport processes and the mode of action of bacterial protein toxins have accelerated the development of toxin-based cargo-delivery vehicle platforms. This review provides an updated survey of the status of available platforms for targeted delivery of therapeutic cargos, outlining various strategies that have been used to deliver different types of cargo into cells. Particular emphasis is placed on the application of toxin-based approaches, examining critical issues that have hampered realization of post-intoxication antitoxins against botulism.
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Cite this article as:
Wilson A. Brenda and Ho Mengfei, Cargo-Delivery Platforms for Targeted Delivery of Inhibitor Cargos Against Botulism, Current Topics in Medicinal Chemistry 2014; 14 (18) . https://dx.doi.org/10.2174/1568026614666141022094517
DOI https://dx.doi.org/10.2174/1568026614666141022094517 |
Print ISSN 1568-0266 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4294 |
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