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The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome

Department of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, ON M5S 3M2, Canada
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Brain Sci. 2019, 9(2), 32; https://doi.org/10.3390/brainsci9020032
Received: 14 December 2018 / Revised: 30 January 2019 / Accepted: 31 January 2019 / Published: 2 February 2019
(This article belongs to the Special Issue Towards Mechanism-based Treatments for Fragile X Syndrome)
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Abstract

Viral vector-mediated gene therapy has grown by leaps and bounds over the past several years. Although the reasons for this progress are varied, a deeper understanding of the basic biology of the viruses, the identification of new and improved versions of viral vectors, and simply the vast experience gained by extensive testing in both animal models of disease and in clinical trials, have been key factors. Several studies have investigated the efficacy of adeno-associated viral (AAV) vectors in the mouse model of fragile X syndrome where AAVs have been used to express fragile X mental retardation protein (FMRP), which is missing or highly reduced in the disorder. These studies have demonstrated a range of efficacies in different tests from full correction, to partial rescue, to no effect. Here we provide a backdrop of recent advances in AAV gene therapy as applied to central nervous system disorders, outline the salient features of the fragile X studies, and discuss several key issues for moving forward. Collectively, the findings to date from the mouse studies on fragile X syndrome, and data from clinical trials testing AAVs in other neurological conditions, indicate that AAV-mediated gene therapy could be a viable strategy for treating fragile X syndrome. View Full-Text
Keywords: adeno-associated virus; autism spectrum disorders; cerebral spinal fluid; fragile X mental retardation protein; neurodevelopmental disorders; viral vector adeno-associated virus; autism spectrum disorders; cerebral spinal fluid; fragile X mental retardation protein; neurodevelopmental disorders; viral vector
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Hampson, D.R.; Hooper, A.W.M.; Niibori, Y. The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome. Brain Sci. 2019, 9, 32.

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