Gene Therapy

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.

Meet Our Team

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.

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.

• Casgevy (Vertex Pharmaceuticals & CRISPR Therapeutics) is a groundbreaking CRISPR-based gene-editing therapy approved for individuals with sickle cell disease or transfusion-dependent beta-thalassemia. It works by editing the patient’s own stem cells to produce healthy red blood cells. The therapy involves collecting stem cells, modifying them in a lab and re-infusing them after the patient undergoes conditioning chemotherapy.
• Elevidys (Sarepta Therapeutics) was developed by researchers and scientists at Nationwide Children's for children with Duchenne muscular dystrophy (DMD) who have a genetically confirmed disease causingvariantsin the DMD gene. The therapy delivers a shortened version of the dystrophin gene to muscle cells via a single intravenous (IV) infusion, aiming to help slow disease progression and improve muscle function.
• Zolgensma (Novartis Gene Therapies) is a one-time gene therapy developed by researchers and scientists at Nationwide Children's to treat spinal muscular atrophy (SMA) in infants and children under age 2 years. It works by replacing the missing or nonfunctioning SMN1 gene with a functional copy, delivered through a single IV infusion. This early intervention helps preserve motor function and prevent progression of the disease. With early in life treatment, patients with SMA can achieve motor development and milestones beyond what was ever observed in the natural course of the disease. Treatment with Zolgensmahas led to ventilator free survival, especially for those who have a predicted severe form of disease, and is truly lifesaving and transforming health and development.
• Hemgenix (CSL Behring) Hemgenix is a gene therapy for adults with hemophilia B that aims to reduce or eliminate the need for regular factor IX infusions. It uses an adeno-associated virus (AAV) vector to deliver a working gene to the liver, allowing the body to produce its own clotting factor. Administered as a single intravenous infusion, Hemgenix offers long-term potential to manage bleeding episodes.
• Kymriah (Novartis) Kymriah is a CAR T-cell therapy designed for children and young adults with certain types of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). It involves collecting a patient’s T cells, genetically modifying them to attack cancer cells, and then reinfusing them. This highly personalized immunotherapy can provide a new option when other treatments have failed.
• Roctavian (BioMarin Pharmaceutical) Roctavian is a gene therapy approved for adults with severe hemophilia A who do not have antibodies against the therapy’s viral vector. Delivered through a single IV infusion, it introduces a functioning gene that enables the body to produce factor VIII, significantly reducing bleeding episodes and the need for routine infusions.
• Zynteglo (Bluebird Bio) is a gene therapy for people with transfusion-dependent beta-thalassemia (TDT). It works by inserting a functional copy of the beta-globin gene into the patient’s stem cells, allowing them to produce healthy hemoglobin. After chemotherapy conditioning, the modified cells are infused back into the patient to reduce or eliminate the need for chronic blood transfusions.
• Lyfgenia (Bluebird Bio) is a gene therapy for sickle cell disease in patients ages 12 years and older with a history of vaso-occlusive events. It uses a lentiviral vector to insert a modified gene into the patient’s stem cells, which are then returned to the body after chemotherapy conditioning. The therapy helps the body produce functional hemoglobin, aiming to reduce painful crises and long-term complications.

Why Choose Us

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

Patient Stories 

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.