Therapeutic approaches for shankopathies


Journal Article

Despite recent advances in understanding the molecular mechanisms of autism spectrum disorders (ASD), the current treatments for these disorders are mostly focused on behavioral and educational approaches. The considerable clinical and molecular heterogeneity of ASD present a significant challenge to the development of an effective treatment targeting underlying molecular defects. Deficiency of SHANK family genes causing ASD represent an exciting opportunity for developing molecular therapies because of strong genetic evidence for SHANK as causative genes in ASD and the availability of a panel of Shank mutant mouse models. In this article, we review the literature suggesting the potential for developing therapies based on molecular characteristics and discuss several exciting themes that are emerging from studying Shank mutant mice at the molecular level and in terms of synaptic function. © 2013 Wiley Periodicals, Inc.

Full Text

Duke Authors

Cited Authors

  • Wang, X; Bey, AL; Chung, L; Krystal, AD; Jiang, YH

Published Date

  • February 1, 2014

Published In

Volume / Issue

  • 74 / 2

Start / End Page

  • 123 - 135

Electronic International Standard Serial Number (EISSN)

  • 1097-4695

International Standard Serial Number (ISSN)

  • 1932-8451

Digital Object Identifier (DOI)

  • 10.1002/dneu.22084

Citation Source

  • Scopus