The promising trajectory of autism therapeutics discovery

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Pharmacological interventions for neurodevelopmental disorders are increasingly tractable. Autism is a neurodevelopmental disorder that affects approximately 1% of the population. Currently, the standard of care is early behavioral therapy. No approved medical treatments for the diagnostic symptoms are available. Strong evidence for genetic causes of autism implicates proteins that mediate synaptic transmission and structure. Mouse models with targeted mutations in these synaptic genes display behavioral symptoms relevant to the social communication abnormalities and repetitive behaviors that define autism spectrum disorder (ASD), along with biological abnormalities in synaptic physiology and morphology. As we discuss here, promising pharmacological targets, emerging from the mouse model studies, are now being pursued in early clinical trials. Thus, a high-prevalence disorder that was previously considered to be medically untreatable is now moving into the therapeutic arena.

Original languageEnglish (US)
Pages (from-to)838-844
Number of pages7
JournalDrug Discovery Today
Volume19
Issue number7
DOIs
StatePublished - 2014

Fingerprint

Autistic Disorder
Pharmacology
Behavioral Symptoms
Standard of Care
Secondary Prevention
Synaptic Transmission
Communication
Clinical Trials
Mutation
Therapeutics
Population
Genes
Proteins
Neurodevelopmental Disorders
Autism Spectrum Disorder

ASJC Scopus subject areas

  • Drug Discovery
  • Pharmacology

Cite this

The promising trajectory of autism therapeutics discovery. / Silverman, Jill L; Crawley, Jacqueline.

In: Drug Discovery Today, Vol. 19, No. 7, 2014, p. 838-844.

Research output: Contribution to journalArticle

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