Center for Personalized Medicine
Programs and Areas of Focus
There are more than 7,000 known genetic diseases, many of which affect infants and children and can cause life-long disability. There are cures for only a handful of these diseases. The goal of the Primary Children’s Center for Personalized Medicine is to transform this critical need for treatments through research, discovery, and innovative therapies.
- Allows critically-ill children to receive personalized, targeted therapies based on their genetic make-up.
- State-of-the-art technology and rapid whole-genome sequencing quickly identify genetic causes of hard-to-diagnose diseases.
- Getting a diagnosis and treatment quickly greatly improves a child's prospects for leading a healthier life.
- Initial efforts are focused on providing answers to critically-ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.
Gene Therapies and Novel Therapeutics
- Enables children with previously debilitating and fatal genetic diseases to survive and thrive.
- Primary Children's is one of only six hospitals nationwide to provide gene therapy for spinal muscular atrophy, one of the most common childhood genetic conditions.
- Clinical trials are testing gene therapy treatments for Duchenne's Muscular Dystrophy, Adrenoleukodystrophy, and other serious diseases.
- Development is underway for novel therapeutics that target specific diseases and improve health.
Stem Cell, Immunologic and Regenerative Medicine
- Uses a child's own cells, or genetically modifies a child's cells and immune system, to fight disease and promote healing.
- Clinical trials are testing the use of stem cells in repairing diseased hearts and other tissues. Research is aimed at developing immunotherapy as a tool to fight pediatric brain tumors.
Universe of Expertise
We are committed to operating a collaborative and coordinated advanced center for genomic research. Our partnership affords us a unique community of doctors and researchers with unmatched knowledge and expertise to continue to discover and develop new cures. No other place can connect clinical, genetic, and familial information like the Primary Children's Center for Personalized Medicine. We offer:
- Exceptional, quality physicians and staff
- Proprietary databases
- Unparalleled facilities
- Perpetual resource-generation and expertise-building
- Robust infrastructure for researchers to tap into
- Sequencing and population databases that produce the most advanced outcomes