The ASPIRE Path in Molecular Medicine

I am a second year pharmacology graduate student in Jeff Conn’s lab. My thesis project is focused identifying and characterizing new therapeutic drug targets for Rett Syndrome. Rett Syndrome is a neurodevelopmental disorder that is characterized by autism-like symptoms, loss of language and motor skills, cognitive impairments, seizures, and stereotyped behaviors. It is a disorder that affects young girls, and currently there are no pharmacological treatments. My lab primarily uses small-molecule drugs called allosteric modulators to fine-tune the function of different metabotropic glutamate receptors, the main glutamate-sensing g protein coupled receptors in the brain, to alter glutamate signaling and provide therapeutic benefit in many neurological disorders. As such, I have also chosen to investigate if modulation of different metabotropic glutamate receptors can improve symptoms in Rett Syndrome.

My clinical interests focus largely on childhood neurodevelopmental disorders, since Rett Syndrome affects young girls in childhood. I am particularly interested in observing how children with Autism or other neurodevelopmental disorders present in clinic, how they interact with their family members, and what the clinical course of action is for these types of children. My ability to shadow my clinical mentor as she interacts with these patients will be invaluable to me as I try to model human Rett Syndrome using mouse behavioral models. I believe that my experience in the clinic and interacting with patients will give me a better understanding of how the patient’s symptoms affect them. This will allow me to critically choose my behavioral models and determine if modulation of metabotropic glutamate receptors is beneficial in improving symptoms of Rett Syndrome.