A novel combination treatment for fragile X syndrome predicted using computational methods.

Wayne Chadwick¹, Ivan Angulo-Herrera¹, Patricia Cogram^2,3, Robert J M Deacon², Daniel J Mason¹, David Brown¹, Ian Roberts¹, Daniel J O'Donovan¹, Michael R Tranfaglia⁴, Tim Guilliams¹, Neil T Thompson¹

¹Healx Ltd., Cambridge, CB3 0DU, UK.

²Department of Genetics, Faculty of Science, Institute of Ecology and Biodiversity (IEB), University of Chile, Santiago 7800024, Chile.

³Center for Neural Circuit Mapping, UCI, School of Medicine, University of California, Irvine, CA 92617, USA.

⁴FRAXA Research Foundation, Newburyport, MA 01950, USA.

PubMed PMC DOI PDF

Abstract

Fragile X syndrome is a neurodevelopmental disorder caused by silencing of the fragile X messenger ribonucleotide gene. Patients display a wide spectrum of symptoms ranging from intellectual and learning disabilities to behavioural challenges including autism spectrum disorder. In addition to this, patients also display a diversity of symptoms due to mosaicism. These factors make fragile X syndrom…

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