Genetics & Precision Medicine Research
Powering Personalized Pediatric Care
Many childhood conditions start at the molecular level. Small changes in DNA, gene expression, or cellular signaling can influence how organs develop, how the immune system responds, and how a child metabolizes medication.
Genetics and precision medicine research at Nemours Children’s focus on decoding those mechanisms. We uncover the biological drivers of rare disorders, aggressive cancers, immune dysfunction, congenital heart disease, and critical illness. Our teams develop more precise diagnostic tools and targeted therapies for children. This approach means care is guided by the underlying biology unique to each child.
Key Areas of Research
Gene Therapy & Genetic Disease Mechanisms | Developing therapies designed to repair or replace disease-causing genes in rare bone disorders, neuromuscular diseases, congenital heart disease, and other complex conditions.
Cancer Genomics & Immune Engineering | Using tumor sequencing and engineered immune cells to design targeted treatments for aggressive leukemias and solid tumors.
Precision Medication & Pharmacogenomics | Applying validated genetic testing to guide medication selection and dosing so children receive safer, more effective treatment across respiratory, gastrointestinal, behavioral, and other specialties.
Molecular Diagnostics & Early Detection | Advancing genomic testing and blood-based biomarkers to diagnose inherited conditions, transplant rejection, autoimmune disease, and neurodevelopmental disorders earlier.
Cellular Energy & Critical Illness Biology | Studying mitochondrial dysfunction and cellular signaling pathways to better predict organ failure and personalize intensive care.
Precision Infrastructure & Integration
Our precision infrastructure brings together pediatric biobanking, advanced sequencing, CLIA-certified molecular diagnostics, specialized cell and tissue cores, and enterprise data analytics into a shared research platform. This structure supports large-scale genomic analysis and focused research into how disease develops at the cellular level.
Integrated within clinical programs, these capabilities move discoveries directly into clinical trials and patient care. The result is stronger diagnostics, more targeted therapies, and faster translation for children with complex conditions.
Genetics & Precision Medicine Clinical Trials
Our investigators lead and participate in gene therapy, cellular immunotherapy, pharmacogenomic-guided treatment, and biomarker validation trials. We collaborate with national research networks to expand access to innovative therapies for children with rare and complex conditions.
Clinical research gives families access to emerging treatments while helping shape the future of pediatric medicine.
Where Genetics & Precision Medicine Research Happens
Genetics and precision medicine research at Nemours is supported by shared laboratory and data resources in Delaware Valley and Florida. These teams collaborate across departments to support both foundational discovery and applied clinical research.
This structure provides specialty programs access genomic testing, molecular analysis, and data expertise. It creates a consistent platform for precision research across our system.
Related Research
Cardiovascular Services Department Research, Florida
FLORIDA
Our researchers use imaging, data analysis, and molecular research to develop new approaches to prevent, diagnose, and treat congenital and acquired heart conditions in children.
Center for Clinical Diagnostics
DELAWARE VALLEY
We provide diagnostic testing services that support clinicians and researchers in diagnosing and monitoring pediatric diseases more effectively.
Center for Pharmacogenomics & Translational Research
DELAWARE VALLEY & FLORIDA
We study how genetics influences medication response in children to develop personalized treatments for conditions like depression and respiratory and gastrointestinal disorders.
Critical Care Mitochondrial Unit
DELAWARE VALLEY
Investigators in our lab study how mitochondrial function affects critically ill children to improve recovery and guide personalized treatments.
Molecular Diagnostic Lab
RESEARCH LAB
Our lab provides molecular testing for genetic conditions, as well as genotyping for pharmacogenomics, allowing personalized drug choice and dosing.
Nemours Cardiac Center for Research & Innovation
DELAWARE VALLEY
We study the impact of innovations, access, and health care delivery on outcomes in heart surgery, preventive cardiology, and more.
Nemours Center for Cancer and Blood Disorders Research
DELAWARE VALLEY & FLORIDA
Our investigators advance pediatric cancer care through innovative research, multi-state clinical trials, and specialized programs in Delaware and Florida.
Neuromuscular Research, Central Florida
CENTRAL FLORIDA
Our team studies neuromuscular diseases in children, focusing on early detection, genetic causes, and strategies to slow disease progression.
Orthopedics Department Research, AI duPont Campus
DELAWARE VALLEY
We’re nationally recognized for leading pediatric orthopedic research, with high-impact publications and a strong presence at major scientific meetings.
Pediatric Tumor Research Lab
CENTRAL FLORIDA
We develop new treatments for challenging childhood cancers, including viral therapies that target tumors while sparing healthy cells for better outcomes.
Pharmacogenetics Laboratory
NORTH FLORIDA
We identify genetic markers that help physicians select the right medications and doses for each child's genetic profile, improving treatment effectiveness while reducing side effects.
Skeletal Dysplasia Lab
DELAWARE VALLEY
Our lab is developing innovative gene therapies and treatments for rare skeletal disorders, specializing in Morquio A syndrome and other inherited conditions.
T Cell Immunology & Immunotherapy Lab
DELAWARE VALLEY
Our lab develops CAR-T cell therapies and identifies new immune targets to advance treatment for pediatric cancers and immune disorders.
Tissue Engineering & Regenerative Medicine Lab
DELAWARE VALLEY
We combine engineering and medicine to create innovative solutions for children with cerebral palsy and heart conditions, improving diagnosis and surgical care.
Genetics & Precision Medicine Clinical Care
Allergy & Immunology
CLINICAL CARE
Coordinated, multidisciplinary care for children who need life-saving organ transplantation, with teams guiding families through evaluation, surgery, and long-term follow-up.
Cardiac Center, Delaware Valley
CLINICAL CARE
Pediatric cardiology, heart surgery, and transplantation at Nemours Children’s Hospital, Delaware and throughout Greater Philadelphia.
Cardiac Center, Florida
CLINICAL CARE
Pediatric cardiology and specialty care at Nemours Children’s Hospital, Florida and throughout Northeast, Northwest, and Central Florida.
Genetics
CLINICAL CARE
Genetic evaluation, diagnosis, and counseling for children with inherited conditions throughout the Delaware Valley and Florida.
Nemours Center for Cancer and Blood Disorders
CLINICAL CARE
Center for Cancer and BD: Comprehensive care for children delivered by multidisciplinary teams across Nemours Children’s hospitals and locations in Delaware Valley and Florida.
Neuromuscular Program
CLINICAL CARE
Certified by the Muscular Dystrophy Association (MDA), Nemours specialists help kids with neuromuscular disorders live their best lives.
Orthopedic Center, Delaware Valley
CLINICAL CARE
Pediatric orthopedic care including neuromuscular, spine, and limb conditions at Nemours Children’s Hospital, Delaware and throughout Greater Philadelphia.
Orthopedic Center, Florida
CLINICAL CARE
Pediatric orthopedic care for bone, joint, and movement conditions at Nemours Children’s Hospital, Florida and throughout North and Central Florida.
Pediatric Critical Care
CLINICAL CARE
Critical care for children with serious illness or injury in specialized units at our Delaware and Florida children's hospitals.
Solid Organ Transplant Program
CLINICAL CARE
Coordinated, multi-specialty care for children who need organ transplantation, with teams guiding families through evaluation, surgery, and long-term follow-up.