期刊
NATURE MEDICINE
卷 19, 期 6, 页码 685-694出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nm.3193
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资金
- Institut Pasteur
- Bettencourt-Schueller foundation
- Centre National de la Recherche Scientifique
- University Paris Diderot
- Agence Nationale de la Recherche [ANR-08-MNPS-037-01-SynGen]
- Neuron-ERANET (EUHF-AUTISM)
- Fondation de France
- Conny-Maeva Charitable Foundation
- Cognacq Jay Foundation
- Orange Foundation
- Fondamental Foundation
- Innovative Medicines Initiative Joint Undertaking [115300]
- European Union
- European Federation of Pharmaceutical Industries and Associations
Autism spectrum disorder (ASD) encompasses a range of disorders that are characterized by social and communication deficits and repetitive behaviors. For the majority of affected individuals, the cause of ASD remains unknown, but in at least 20% of the cases, a genetic cause can be identified. There is currently no cure for ASD; however, results from mouse models indicate that some forms of the disorder could be alleviated even at the adult stage. Genes involved in ASD seem to converge on common pathways altering synaptic homeostasis. We propose, given the clinical heterogeneity of ASD, that specific 'synaptic clinical trials' should be designed and launched with the aim of establishing whether phenotype 'reversals' could also occur in humans.
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