Gene Therapy Center of Excellence

Conditions We Treat

Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is a rare condition defined by worsening muscle weakness and damage. It is the most common inherited neuromuscular disorder that affects all races and ethnicities. DMD only affects males and children with DMD may lose the ability to walk as early as 7 years of age.

Spinal Muscular Atrophy

Spinal muscular atrophies (SMA) are a group of genetic (passed down by parents) diseases that affect motor neurons (nerve cells) in the spinal cord, causing the weakening of voluntary muscles (muscles that you control).

Sickle Cell Disease

Sickle cell disease is a lifelong, inherited blood disorder that affects how oxygen flows through the body—causing pain, anemia, and serious complications. Learn about symptoms, treatment options, emergency care, and how to support your child’s health every step of the way.

Hemophilia

Hemophilia is an inherited blood disorder. In hemophilia, a blood clotting factor is missing. In Hemophilia A, Factor VIII (8) is missing. In Hemophilia B, Factor IX (9) is missing. Without these factors, blood will not clot well. People with hemophilia are born with the disorder. It is not contagious.

Leukemia (ALL and AML)

Leukemia (ALL and AML) is the most common form of childhood cancer. It affects the tissues of the body which make the blood cells and the bone marrow. When leukemia strikes, the body makes an abundance of abnormal white cells that invade the marrow and crowd out the normal healthy blood cells.

A Dedicated Team

At Nationwide Children’s Hospital, gene and cell therapy isn’t an add-on service; it is a specialty focus just like cancer, transplantation, neuromuscular or hematology. Families come here because of our team whose entire focus is just successfully delivering gene and cell therapy, from the very first appointment through long-term follow-up. The decision to proceed with a gene replacement therapy is likely to be one of the biggest decisions for your family and in your child’s care. We provide expert and detailed care bringing world class expertise in gene and cell therapies, compassionate and holistic care, vigilant follow up, and close communication with you and throughout the care team to deliver these precision therapies safely. We partner with you and your healthcare team every step of the way, providing education so you can feel assured and equipped in the care and management before and after your gene therapy day. Your journey does not end with the dosing day and will require detailed and frequent safety monitoring coordinated by the Gene Therapy Center of Excellence clinical team. A multidisciplinary team included dedicated hematology, gastroenterology/ hepatology, cardiology and other services allows us to readily provide any additional care and monitoring that may be needed following gene therapy. That means your child is supported by a team of specialists who dedicate their careers to this field, providing the depth of experience and attention you won’t find everywhere else.

For questions please call our team at (614) 938-1107

Meet Our Leaders

Kevin M. Flanigan

Kevin M. Flanigan, MD, is an attending neurologist at Nationwide Children's and director of the Jerry R. Mendell Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital.

Diana X. Bharucha-Goebel

Diana Bharucha-Goebel, MD, is Medical Director of the Gene Therapy Center of Excellence and principal investigator in the Jerry R. Mendell Center for Gene Therapy.

Patient Stories

Wilson Family Story

Kael and Moci were adopted from an orphanage in China by their parents Mandy and Chris. Both have beta thalassemia major, a genetic blood disorder. Since being adopted, Kael and Moci have had blood transfusion appointments at Nationwide Children's every three weeks, which last six to eight hours. The siblings also take daily medication to counter the side effects of frequent blood transfusions. This past summer, Kael and Moci made Nationwide Children’s Hospital history as our first patients to receive gene therapy for beta thalassemia. So far, the therapy has been a success: both kids have gone three months without a blood transfusion, and their hemoglobin levels are climbing. They were quickly cleared to return to school and their regular activities a month after discharge!

Meet Gideon

In a powerful milestone for pediatric medicine, 5-year-old Gideon Griffiths became the first patient to receive a groundbreaking gene therapy for Duchenne muscular dystrophy—developed and tested at Nationwide Children’s Hospital. This life-changing treatment, now FDA-approved, offers new hope to families across the country and marks Nationwide Children’s second gene therapy to reach this historic achievement.

Meet Evelyn

“Something like this has never been achieved before.” – Jerry Mendell, MD Spinal Muscular Atrophy Type 1 is the leading genetic cause of infant death. But for Evelyn Villarreal, gene therapy changed everything. After losing their first daughter to SMA, Evelyn’s parents feared history would repeat itself—until they found hope at Nationwide Children’s Hospital. Evelyn became one of the first children to receive a pioneering gene therapy, and today, she’s thriving. This landmark achievement marks the first time gene therapy has been successfully applied to a neuromuscular disease, offering families a future once thought impossible.

Meet Jackson

Five years ago, Jackson was a boy who ran, played sports, and walked his dog. Today, Duchenne Muscular Dystrophy has taken his ability to walk—but not his spirit. With the help of his care team at Nationwide Children’s Hospital, Jackson completed a marathon in his wheelchair, pushed by the very people fighting to end the disease. Every mile, every step, every dollar raised fuels groundbreaking research to change the future for Jackson and all boys with Duchenne. Because while Jackson can’t run—we can.

Meet Easton and Connor

At just five years old, Connor Stoll became the first patient to receive a groundbreaking gene therapy for Duchenne muscular dystrophy at Nationwide Children’s Hospital. Five years later, Connor is thriving—climbing stairs, playing baseball, and keeping up with his friends. For Easton Reed, the therapy brought similar transformation. Just days after his infusion, he was racing up stairs for the first time in his life.

Research and Clinical Trials

The Jerry R. Mendell Center for Gene Therapy aims to investigate and employ the use of gene and cell-based therapeutics for prevention and treatment of human diseases. Nationwide Children's has over 25 open and enrolling trials for cell and gene therapies. See the links below for the latest studies and eligibility.

• Gene Therapy Studies
• Cell Therapy Studies
• Center for Gene Therapy

Therapies

The Gene Therapy Center of Excellence has dosed over 100 therapies since it's inception. We are proud to have been the leading institutions to discover and develop two of the currently approved gene replacement therapies, Elevidys and Zolgensma.

View Available Therapies