CHOC Children’s Research Institute Helping Transform Pediatric Medicine

Research Lab Technician with MicroscopeUpdate from Brent Dethlefs, executive director, CHOC Children’s Research Institute

The CHOC Children’s Research Institute is proud of the work our physicians and researchers are doing to advance pediatric medicine, improve clinical outcomes and save young lives. Over the past few years, CHOC clinicians have introduced a new procedure that cures chronic irregular heartbeats without radiation, utilized the latest genomic sequencing techniques to change the lives of children with cancer, and launched a $3 million study funded by the NIH to promote research around the country in search of the underlying causes of autism. In 2013, we enrolled over 300 additional child and adolescent participants in our nearly 500 on-going open and active clinical research studies.

In the coming year, we are looking forward to enhancing translational research collaboration through implementation of the CHOC Children’s – UC Irvine Child Health Research strategic plan to understand, among other things, the pediatric origins of adult diseases. We are also looking forward to strengthening our participation in national cystic fibrosis (CF) research that is – for the first time – treating and perhaps curing the underlying cause of this devastating disease for a sub-set of CF patients; continuing to advance genomic and RNA sequencing to individualize treatment for childhood cancer; continuing to serve the autism community by contributing stem cells derived from the skin of autism patients to promote research into the underlying causes of this baffling spectrum of disorders; and installing a GMP-compliant cell manufacturing facility to test the ability of laboratory-derived neural stem cells to treat the severe neurological deficits found in lysosomal storage disorders, which are inherited fatal diseases diagnosed in young children as a result of an enzyme deficiency.

Our team’s steadfast commitment to the health and well being of children will continue to drive our efforts to unravel the complex underpinnings of diseases that affect pediatric patients. This dedication will keep CHOC at the forefront of technology, science and innovation, transforming the delivery of pediatric medicine.

For more information about the CHOC Children’s Research Institute, visit or call 714-509-4341.

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