Abstract
In many neurological disorders strategies for a specific delivery of a biological activity from the periphery to the central nervous system (CNS) remains a considerable challenge for successful therapy. Reporter assays have established that the non-toxic C-fragment of tetanus toxin (TTC), provided either as protein or encoded by non-viral naked DNA plasmid, binds pre-synaptic motor neuron terminals and can facilitate the retrograde axonal transport of desired therapeutic molecules to the CNS. Alleviated symptoms in animal models of neurological diseases upon delivery of therapeutic molecules offer a hopeful prospect for TTC therapy. This review focuses on what has been learned on TTC-mediated neuronal targeting, and discusses the recent discovery that, instead of being merely a carrier molecule, TTC itself may well harbor neuroprotective properties.
Highlights
Neurodegenerative disorders are a group of pathological conditions that typically originate from progressive dysfunction and loss of neurons or synaptic contacts in defined areas of the nervous system
Worthwhile to look for delivery methods that are minimally invasive and can be administered to the peripheral tissues but which may bear the therapeutic effect via distal neurons into the central nervous system (CNS)
This review focuses on what has been learned about tetanus toxin C-fragment (TTC) based therapies with special emphasis on motor neuron diseases
Summary
Neurodegenerative disorders are a group of pathological conditions that typically originate from progressive dysfunction and loss of neurons or synaptic contacts in defined areas of the nervous system. Normally precludes direct vascular delivery of large molecules to the CNS It is, worthwhile to look for delivery methods that are minimally invasive and can be administered to the peripheral tissues but which may bear the therapeutic effect via distal neurons into the CNS. These methods have relied on the ability of neurotropic viruses to serve as carriers, because they are taken up by the nerve termini and transported to the soma. Besides the previously mentioned neurodegenerative conditions, the design of enzyme-replacement therapies for lysosomal storage disorders with neurological involvement may benefit from the trans-synaptic properties of TTC
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