Close
Enter your
text here

Gene and Cell Therapy at Nationwide Children’s: Leading Discovery, Driving Care

Gene and Cell Therapy at Nationwide Children’s: Leading Discovery, Driving Care 1024 572 Nationwide Children's
artistic illustration of adenovirus on a dark purplish background

Nationwide Children’s has been a pioneer in gene and cell therapy for more than 30 years. Since our first clinical trial in 1999, our team has only accelerated the pace of innovation, discovery and patient-centered care.

On this page, you’ll find a collection of stories featuring our advances and our researchers. Because behind every breakthrough is a team of scientists, patients and their families, advocates, regulators and more driving the work forward, these stories embrace the ecosystem of discovery here at Nationwide Children’s.

Universal Donor CAR NK Cells: A New Platform Technology for Cancer

artistic rendering of an NK cell on a faded blue background with other cells faintly visible in the background
Making Universal Donor CAR NK Cells
Universal Donor CAR NK Cells: A New Platform Technology for Cancer
Startups Update: Where Are They Now?

Vironexis: Breaking New Ground in Cancer Immunotherapy

Breaking New Ground in Cancer Immunotherapy
A Single-Dose Cancer Immunotherapy via Gene Transfer
Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells

On the Road to Gene Therapy for Cystic Fibrosis

Genome Database Analysis Suggests Cystic Fibrosis is More Globally Distributed Than Previously Believed
Beyond Modulators: Ensuring All Patients With Cystic Fibrosis Benefit From the Next Wave of Therapy

Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease

Mother and son sitting in hospital bed with doctors Allison Bradbury and Emily de los Reyes standing at the bedside. Everyone is excited; soon the child will receive a brand new gene therapy for his ultrarare disease.
Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease

Exon-Skipping Gene Therapy in Duchenne Muscular Dystrophy

Proof-of-Concept Study Shows Promise of Exon-Skipping Gene Therapy Approach in Duchenne Muscular Dystrophy

Improving Viral Vector Manufacturing Safety

Clean Room
Novel Viral Vector Manufacturing Approach Could Improve Safety of Gene Therapy

Making Gene Therapy Accessible

Gene Therapy for the Masses?

Meet Kael, Moci and the Rest of the Wilson Family

Watch the rest of the Kael and Moci’s story following their journey to get gene therapy for Beta Thalassemia and see more videos highlighting gene and cell therapy work at Nationwide children’s: Changing Lives Through Gene Therapy

A Platform Technology for Protein Folding Disorders

Dr. Zarife Sahenk, MD, PhD
One Gene, Big Impact: The Science Behind a New Therapy for Brain and Muscle Health

Cracking the Code: Dual Gene Therapy

Has the Next Generation of Gene Therapy Arrived?

The First FDA-Approved Systemic Gene Therapy 

Colorful illustration of gene therapy in action
FDA APPROVAL: Gene Therapy Comes of Age

Microdystrophin Gene Therapy for DMD

FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Featured Researchers

Click the names below to learn more about each of our featured researcher’s work and career. To learn more about the whole team, visit: Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s

Allison Bradbury, MS, PhD, principal investigator in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s

portrait of Sriram Vaidyanathan, PhD

Sriram Vaidyanathan, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s

Nizar Y. Saad, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s

Scott Harper, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s

You might also like