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Gene Therapy for Misfolding Protein Diseases of the Central Nervous System

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  • Published:
Neurotherapeutics

Abstract

Protein aggregation as a result of misfolding is a common theme underlying neurodegenerative diseases. Accordingly, most recent studies aim to prevent protein misfolding and/or aggregation as a strategy to treat these pathologies. For instance, state-of-the-art approaches, such as silencing protein overexpression by means of RNA interference, are being tested with positive outcomes in preclinical models of animals overexpressing the corresponding protein. Therapies designed to treat central nervous system diseases should provide accurate delivery of the therapeutic agent and long-term or chronic expression by means of a nontoxic delivery vehicle. After several years of technical advances and optimization, gene therapy emerges as a promising approach able to fulfill those requirements. In this review we will summarize the latest improvements achieved in gene therapy for central nervous system diseases associated with protein misfolding (e.g., amyotrophic lateral sclerosis, Alzheimer’s, Parkinson’s, Huntington’s, and prion diseases), as well as the most recent approaches in this field to treat these pathologies.

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Acknowledgments

This work was supported, in part, by NIH grant R01NS073940 to KSB.

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  1. Department of Neurological Surgery, University of California San Francisco, 1855 Folsom Street, San Francisco, CA, USA

    Waldy San Sebastian, Lluis Samaranch, Adrian P. Kells, John Forsayeth & Krystof S. Bankiewicz

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  2. Lluis Samaranch
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Correspondence to Krystof S. Bankiewicz.

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San Sebastian, W., Samaranch, L., Kells, A.P. et al. Gene Therapy for Misfolding Protein Diseases of the Central Nervous System. Neurotherapeutics 10, 498–510 (2013). https://doi.org/10.1007/s13311-013-0191-8

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