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Therapeutic approaches for shankopathies
Author(s) -
Wang Xiaoming,
Bey Alexandra L.,
Chung Leeyup,
Krystal Andrew D.,
Jiang YongHui
Publication year - 2014
Publication title -
developmental neurobiology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.716
H-Index - 129
eISSN - 1932-846X
pISSN - 1932-8451
DOI - 10.1002/dneu.22084
Subject(s) - biology , neuroscience , autism , autism spectrum disorder , gene , computational biology , genetics , psychology , developmental psychology
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. Develop Neurobiol 74: 123–135, 2014