Viral vector-mediated gene transfer for CNS disease

Expert Opinion on Biological Therapy
Brooke R SnyderThais Federici

Abstract

Gene therapy is a promising strategy for the treatment of many neurological disorders that currently lack effective treatment. Recent improvements in vectorology and vector engineering have improved overall safety and delivery of viral vectors. This review discusses the current state of viral vector development and clinical use, as well as routes of delivery, and clinical trials for neurological disorders. Viral vectors may be delivered directly or remotely to the CNS, largely depending on the nature of the disease and the tropism of the vector. Nonetheless, delivery remains one of the major limitations of successful gene transfer to the CNS. Although the majority of clinical trials have centered on gene replacement and neuroprotection approaches, the field is advancing in the direction of neuromodulation, gene silencing and other newer strategies.

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Citations

Mar 10, 2011·Journal of Speech, Language, and Hearing Research : JSLHR·Mark A Parker
Jul 19, 2011·Neurology Research International·Chalonda R HandyThais Federici
Sep 6, 2011·Neurobiology of Disease·Thais Federici, Nicholas M Boulis
Jul 23, 2013·European Journal of Pharmacology·Fred de WinterJoost Verhaagen
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Oct 4, 2011·Pharmaceuticals·Benjamin Kopecky, Bernd Fritzsch
Apr 9, 2013·Neurological Research·Hanieh JalaliMasoud Soleimani
Dec 30, 2011·Clinical Pharmacology and Therapeutics·D R HampsonL K K Pacey

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