Gene expression in blood is associated with risperidone response in children with autism spectrum disorders

L. Lit, F. R. Sharp, K. Bertoglio, B. Stamova, B. P. Ander, A. D. Sossong, R. L. Hendren

Research output: Contribution to journalArticlepeer-review

21 Scopus citations


Children with autism spectrum disorders (ASDs) often have severe behavioral problems. Not all children with these problems respond to atypical antipsychotic medications; therefore, we investigated whether peripheral blood gene expression before treatment with risperidone, an atypical antipsychotic, was associated with improvements in severe behavioral disturbances 8 weeks following risperidone treatment in 42 ASD subjects (age 112.7±51.2 months). Exon expression levels in blood before risperidone treatment were compared with pre-post risperidone change in Aberrant Behavior Checklist-Irritability (ABC-I) scores. Expression of exons within five genes was correlated with change in ABC-I scores across all risperidone-treated subjects: GBP6, RABL5, RNF213, NFKBID and RNF40 (α<0.001). RNF40 is located at 16p11.2, a region implicated in autism and schizophrenia. Thus, these genes expressed before treatment were associated with subsequent clinical response. Future studies will be needed to confirm these results and determine whether this expression profile is associated with risperidone response in other disorders, or alternative antipsychotic response within ASD.

Original languageEnglish (US)
Pages (from-to)368-371
Number of pages4
JournalPharmacogenomics Journal
Issue number5
StatePublished - Oct 2012


  • atypical antipsychotic
  • autism
  • gene expression
  • risperidone

ASJC Scopus subject areas

  • Pharmacology
  • Molecular Medicine
  • Genetics


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