CHOP Research Institute

Center for Applied Genomics

Today's Research Becomes Tomorrows Cure

CAG is at the forefront of efforts to identify genes implicated in common childhood psychiatric disorders. We were the first group to identify common gene variants that increase the risk of developing autism spectrum disorders, and we have identified a series of genetic variants that are involved in neurodevelopment.

A number of research projects in development aim to provide more effective treatment for neurological and psychiatric disorders. These include targeted programs to treat children diagnosed with ADHD, autism spectrum disorders, epilepsy, and Rett syndrome.

For each of these disorders, the most effective approach may be to sequence the genomes of affected children. As the only pediatric center with large-scale genome sequencing technology, CAG is uniquely equipped to fulfill this ambitious program.

By sequencing genes that associate with these disorders, we may be able to deliver medications specifically tailored to a child's genomic profile - an approach known as personalized medicine.

This is possible because every drug that has reached the stage of clinical trials has an existing literature, which outlines the molecular properties of the drug and clues as to its effects on physiology. By matching the biological function of a damaged gene with the biological properties of an existing medication, we can quickly come up with a targeted treatment.

Often this involves drug repositioning, meaning that we take a medication previously developed to treat one disorder and applying it to an entirely different disorder. For example, we are now working on a program that utilizes a drug originally developed to treat Alzheimer’s and applying instead to treat rare forms of ADHD.

CAG is pioneering the personalized medical model, and has proposed a number of innovative treatments for neurological and psychiatric disorders that are awaiting support.

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