Targeted treatments in autism and fragile X syndrome

C. Kaan Gürkan, Randi J Hagerman

Research output: Contribution to journalArticle

30 Citations (Scopus)

Abstract

Autism is a neurodevelopmental disorder consisting of a constellation of symptoms that sometimes occur as part of a complex disorder characterized by impairments in social interaction, communication and behavioral domains. It is a highly disabling disorder and there is a need for treatment targeting the core symptoms. Although autism is accepted as highly heritable, there is no genetic cure at this time. Autism is shown to be linked to several genes and is a feature of some complex genetic disorders, including fragile X syndrome (FXS), fragile X premutation involvement, tuberous sclerosis and Rett syndrome. The term autism spectrum disorders (ASDs) covers autism, Asperger syndrome and pervasive developmental disorders (PDD-NOS) and the etiologies are heterogeneous. In recent years, targeted treatments have been developed for several disorders that have a known specific genetic cause leading to autism. Since there are significant molecular and neurobiological overlaps among disorders, targeted treatments developed for a specific disorder may be helpful in ASD of unknown etiology. Examples of this are two drug classes developed to treat FXS, Arbaclofen, a GABA B agonist, and mGluR5 antagonists, and both may be helpful in autism without FXS. The mGluR5 antagonists are also likely to have a benefit in the aging problems of fragile X premutation carriers, the fragile X-associated tremor ataxia syndrome (FXTAS) and the Parkinsonism that can occur in aging patients with fragile X syndrome. Targeted treatments in FXS which has a well known genetic etiology may lead to new targeted treatments in autism.

Original languageEnglish (US)
Pages (from-to)1311-1320
Number of pages10
JournalResearch in Autism Spectrum Disorders
Volume6
Issue number4
DOIs
StatePublished - Oct 2012

Fingerprint

Fragile X Syndrome
Autistic Disorder
Therapeutics
GABA-B Receptor Agonists
Asperger Syndrome
Rett Syndrome
Inborn Genetic Diseases
Tuberous Sclerosis
Parkinsonian Disorders
Interpersonal Relations
Communication
Pharmaceutical Preparations
Genes

Keywords

  • Autism
  • Fragile X syndrome
  • GABA
  • mGluR
  • Treatment

ASJC Scopus subject areas

  • Psychiatry and Mental health
  • Clinical Psychology
  • Developmental and Educational Psychology

Cite this

Targeted treatments in autism and fragile X syndrome. / Gürkan, C. Kaan; Hagerman, Randi J.

In: Research in Autism Spectrum Disorders, Vol. 6, No. 4, 10.2012, p. 1311-1320.

Research output: Contribution to journalArticle

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