Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders

  • Dominic J. Gessler
  • Guangping GaoEmail author
Part of the Methods in Molecular Biology book series (MIMB, volume 1382)


Metabolic disorders comprise a large group of heterogeneous diseases ranging from very prevalent diseases such as diabetes mellitus to rare genetic disorders like Canavan Disease. Whether either of these diseases is amendable by gene therapy depends to a large degree on the knowledge of their pathomechanism, availability of the therapeutic gene, vector selection, and availability of suitable animal models. In this book chapter, we review three metabolic disorders of the central nervous system (CNS; Canavan Disease, Niemann–Pick disease and Phenylketonuria) to give examples for primary and secondary metabolic disorders of the brain and the attempts that have been made to use adeno-associated virus (AAV) based gene therapy for treatment. Finally, we highlight commonalities and obstacles in the development of gene therapy for metabolic disorders of the CNS exemplified by those three diseases.

Key words

Gene therapy CNS Metabolic disorders AAV Retrovirus Phenylketonuria Canavan’s disease Niemann–Pick disease 


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Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  1. 1.University of Massachusetts Medical SchoolWorcesterUSA

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