Mechanisms and Models

The goal of this project is to identify potential gene targets for therapeutic intervention in autism; specifically to include:

• describe the behavioral characteristics (phenotypes) of mice with genes highly associated with ASDs (neurexin1, neuorligin3) and identify genetic modifiers.
  
  
• identify and understand the importance of  two pathways (mGLUR and IGF1) in mice that lack genes that have previously been linked to autism (Fmr1, MeCP2, cAMP-GEFII and PTEN)

As gene targets and pathways are identified and understood, we will move quickly to test therapeutic interventions and where possible to clinical trials.  An initial pilot is underway to develop a therapeutic intervention with the MeCP2 gene, using known therapeutics that are currently in use in the pediatric population.  We have initiated discussions with potential partners for testing the intervention in clinical trials.